Tag Archives: Sickle Cell News

GBT and SCDAA Kick Off “Lift Every Voice to Shine the Light on Sickle Cell”

May 04, 2021 at 8:00 AM EDT
SOUTH SAN FRANCISCO, Calif. and HANOVER, Md., May 04, 2021 (GLOBE NEWSWIRE) —In recognition of World Sickle Cell Day, which falls on June 19, 2021,Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) and the Sickle Cell Disease Association of America, Inc. (SCDAA) today launched “Lift Every Voice to Shine the Light on Sickle Cell” – a contest that will feature original spoken word pieces to raise awareness of sickle cell disease (SCD). The contest, which aims to elevate the voices and experiences of people living with SCD and their caregivers, is presented by Sickle Cell Speaks, GBT’s SCD education campaign that highlights authentic stories of those living with SCD to dispel misconceptions about the disease.
“People with sickle cell disease possess an incredibly rich and diverse array of creative abilities, often used to express the physical and emotional burden of living with this complex and devastating disease,” said Beverley Francis-Gibson, M.A., president and CEO of the SCDAA. “Celebrating these voices is critical as we work to overcome the legacy of stigma and misinformation that have a direct impact on health outcomes. We are proud to partner with GBT to shine the light on the challenges that SCD warriors face with strength and resilience.”
Spoken word poetry is an art form rooted in traditions of storytelling to convey compelling messages and personal experiences. People living with SCD and their caregivers are invited to sign up to submit videos of themselves performing original spoken word pieces about their experience with SCD. To learn more about the contest details and how to submit a video, please email patientevents@sicklecellspeaks.info or visit www.facebook.com/SickleCellSpeaks. Participants must sign up by May 24, 2021, and be U.S. residents. For each eligible submission received, GBT will donate $100 to the SCDAA, up to a total donation of$5,000. The contest winner will be featured in a GBT event at the SCDAA Annual Convention in October 2021. “Shine the Light on Sickle Cell” is a collaboration between SiNERGe and SCDAA.
“GBT is proud to partner with SCDAA to recognize the many inspiring voices within the sickle cell community who have rallied for progress in the face of tremendous health and societal challenges over the last year,” said Jung E. Choi, chief business and strategy officer, and head of patient advocacy and government affairs at GBT. “People with SCD suffer from a terrible, life-threatening disease that is made worse by being subjected to racial bias. We reiterate our commitment to shining a brighter light on the inequities these patients encounter and will continue working with all our partners to ensure access to the high-quality care patients deserve.”
Performances by the spoken word contest winner and finalists will be featured in a virtual event on Friday, June 18, 2021, at 4:00 p.m. PT and Saturday, June 19, 2021, at 12:00 p.m. PT on the Sickle Cell Speaks Facebook and Instagram pages. The event will be hosted and feature performances by three SCD advocates who are passionate about using spoken word to educate and inspire change:

  • Charly Richard, musician and writer
  • DeMitrious Wyant, musician and entrepreneur
  • Candis St. John, nurse and poet

About Sickle Cell Disease
Sickle cell disease (SCD) affects an estimated 100,000 people in the United States,1 an estimated 52,000 people inEurope,2 and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa.1 It also affects people of Hispanic, South Asian, Southern European, and Middle Eastern ancestry.1 SCD is a lifelong inherited blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.3 Due to a genetic mutation, people with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled – deoxygenated, crescent-shaped, and rigid.3-5 The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.4-7
About SCDAA
Sickle Cell Disease Association of America(SCDAA) advocates for people affected by sickle cell conditions and empowers community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. The association and more than 50 member organizations support sickle cell research, public and professional health education and patient and community services. Visitwww.sicklecelldisease.org.
About Global Blood Therapeutics
Global Blood Therapeutics(GBT) is a biopharmaceutical company dedicated to the discovery, development, and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta®(voxelotor), the first FDA-approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in development to address pain crises associated with the disease, and GBT021601 (GBT601), the company’s next-generation hemoglobin S polymerization inhibitor. In addition, GBT’s drug discovery teams are working on new targets to develop the next wave of treatments for SCD. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news.
References

  1. Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed June 3, 2019.
  2. European Medicines Agency.https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed June 12, 2020.
  3. National Heart, Lung, and Blood Institute website. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed August 5, 2019.
  4. Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
  5. Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
  6. Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
  7. Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.

GBT Contact Information:
Steven Immergut
650-410-3258
simmergut@gbt.com
SCDAA Contact Information:
Emma Day
eday@sicklecelldisease.org
Kyri Jacobs
kjacobs@sicklecelldisease.org

Download PDF | Visit the GBT Website  

SCDAA partners with Sickle Cell Community Consortium

Sickle Cell Disease Association of America partnered with the Sickle Cell Community Consortium to advocate for legislation benefiting people with sickle cell disease and their families. The partnership includes collaboration on the association’s annual advocacy day initiatives, meetings and trainings and in developing federal legislative priorities.
“Sickle Cell Disease Association of America and the Sickle Cell Community Consortium share the same mission of improving the lives of people with sickle cell disease,” said Beverley Francis-Gibson, president and CEO of the Sickle Cell Disease Association. “We’re excited to work with the consortium, which brings a range of organizations, advocates and advisers that will help us achieve our legislative goals together.”
A nonprofit formed in 2014, the Sickle Cell Community Consortium consists of sickle cell community organizations, patient and caregiver advocates, community partners and medical and research advisers working together to represent people with sickle cell disease. The consortium identifies and implements strategies and partnerships to address needs in the sickle cell community.
Sickle Cell Disease Association of America’s annual advocacy day, supported by the partnership between the association and consortium, generates public awareness of sickle cell disease and kickstarts momentum to push for legislative reform. The day provides training, resources and guidance to participants interested in advocacy work.  

SCDAA Launches New Educational Materials to Support Children’s Blood Transfusion

Sickle Cell Disease Association of America, Inc. (SCDAA) and Hemanext Inc., a privately held medical technology company dedicated to improving the quality, safety, efficacy and cost of red blood cell (RBC) transfusion therapy, today announced the launch of new educational material to help SCDAA deliver on its mission and meet its goals. Hemanext has sponsored the creation of a set of educational materials, one for a child and one for a caregiver, to educate on blood transfusions. This collaboration is part of SCDAA and Hemanext’s partnership, which began in 2020.
Sickle cell disease (SCD) affects 100,000 individuals in the United States, disproportionately affecting African Americans. SCD occurs in about one in 365 Black or African American births.1 The genetic disease is associated with serious, life-threatening complications, including stroke and acute chest syndrome (ACS).2,3 As result, people with SCD often require chronic red blood cell transfusions,2 which for some patients is a life-saving therapy.3
Children and parents may experience anxiety because they are unaware of the process to receive a blood transfusion. The goal of these two educational materials is to inform and empower patients and their caregivers about this important therapy. SCDAA will make these the resources available to the sickle cell community.
“During a review of our currently available educational resources, we identified the opportunity to help young sickle cell warriors prepare for their transfusions,” said SCDAA President and CEO Beverley Francis-Gibson. “We appreciate that Hemanext has stepped up to help us fill this information need and make a difference breaking the sickle cycle.”
“It is a privilege to continue our partnership with Ms. Francis-Gibson and her dedicated team at SCDAA, the premier sickle cell organization,” said Hemanext President and CEO Martin Cannon. “We are committed to helping SCDAA achieve its mission and enhance the lives of members of the sickle cell community.”
“As we enter the second year of our alliance, we will continue to look for ways to support SCDAA, patients, and families during these difficult times,” said Alex Marichal, VP, Marketing, Hemanext. 
ABOUT SCDAA
SCDAA’s mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. Visit www.sicklecelldisease.org.
ABOUT HEMANEXT
Hemanext’s mission is to help patients enjoy healthier lives through safer transfusions. Hemanext’s technology is a processing and storage system that is designed to remove the fuel for oxidative damage to red blood cells. Hemanext is focused on supporting clinicians and healthcare practitioners who prescribe life-saving RBC transfusions to their patients. Visit Hemanext.com to learn more.

  1. Data & Statistics on Sickle Cell Disease. Centers for Disease Control and Prevention. https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed March 19, 2021.
  2. Understanding Sickle Cell Disease. American Society of Hematology, 2019. Available at https://www.hematology.org/education/clinicians/guidelines-and-quality-care/clinical-practice-guidelines/sickle-cell-disease-guidelines. Accessed March 23, 2021.
  3. Chou ST, Fasano RM. Management of Patients with Sickle Cell Disease Using Transfusion Therapy: Guidelines and Complications. Hematology/oncology Clinics of North America. 2016 Jun;30(3):591-608. DOI: 10.1016/j.hoc.2016.01.011.

 

Sickle Cell Disease Association of America and Aruvant Sciences Forge New Partnership to Educate Around Gene Therapy

The Sickle Cell Disease Association of America (SCDAA) and Aruvant Sciences are proud to announce a new partnership to create educational programs to increase awareness of gene therapy as a potential curative treatment option for sickle cell disease patients. This collaboration will help SCDAA continue to deliver on its mission, while assisting Aruvant in learning more about the needs of sickle cell disease (SCD) patients. Under the agreement, Aruvant will collaborate with SCDAA to host local and national educational events and develop materials for a public-awareness campaign.
“In partnership with SCDAA, we are working to educate patients about gene therapy, while gaining critical insights from the patient community for our ARU-1801 SCD development program,” said Will Chou, M.D., chief executive officer (CEO) of Aruvant. “Now is a perfect time to work with SCDAA to educate the community about gene therapy since we have an open and enrolling phase 1/2 clinical trial for our potentially curative experimental gene therapy, ARU-1801.”
Sickle cell disease affects 100,000 individuals in the United States, disproportionately affecting African Americans with one in 500 African Americans suffering from the disease. This inherited disease affects the production of hemoglobin, a protein in red blood cells that carries oxygen throughout the body. The disease occurs when people inherit a mutation from each of their parents which causes people with SCD to not have normal, healthy adult hemoglobin in their red blood cells and instead have an abnormal hemoglobin called sickle hemoglobin. SCD can cause frequent episodes of severe pain, weakness and other serious complications. Fetal hemoglobin is an “anti-sickling” hemoglobin that is present before birth in the red blood cells. After birth, the gene that makes fetal hemoglobin turns off, which mostly stops the production of fetal hemoglobin. More fetal hemoglobin in the blood can mean fewer episodes of sickling and pain.
“In partnership with Aruvant, we can provide the critical education needed for our community to understand gene therapy and how these promising new treatments work to treat and maybe cure this genetic disease that impacts so many in our community,” said Beverley Francis-Gibson, SCDAA president and CEO. “Partnering with companies like Aruvant is critical to help us support the research that could change the lives of many sickle cell disease patients.”
Aruvant and SCDAA’s educational events will review gene therapy and ongoing research, including discussion around Aruvant’s MOMENTUM study. This clinical trial is examining a one-time investigational treatment, ARU-1801, to increase levels of fetal hemoglobin in patients with severe sickle cell disease, with the hope of fewer episodes of sickling and pain. Aruvant provided funding for SCDAA’s 48th Annual National Convention 2020 which begins tomorrow, October 13, and will continue through October 17. To register, please visit https://bit.ly/SCDAA2020Convention.
   

Sickle Cell Disease Association of America Partners with HealthWell Foundation

New Fund Launches to Provide Financial Assistance to People with Sickle Cell Disease
Copayment and Premium Assistance Now Available
(April 15, 2020 – Hanover, MD)  –   The Sickle Cell Disease Association of America is proud to announce its partnership with the HealthWell Foundation®, an independent non-profit that provides a financial lifeline for inadequately insured Americans.  To support the sickle cell community, HealthWell has launched a new fund to provide copayment and premium assistance. Through the fund, HealthWell will provide up to $10,000 in financial assistance for a 12-month grant period to eligible patients who have annual household incomes up to 500 percent of the federal poverty level.
“We are excited that the HealthWell Foundation will provide much needed resources to individuals living with sickle cell disease during this difficult time. I am pleased that they are partnering with SCDAA to support the sickle cell community and reach as many individuals as possible,” says SCDAA President, Beverley Francis-Gibson.
“The HealthWell Foundation is proud to partner with the SCDAA to spread the word about this exciting new fund and to assist people living Sickle Cell Disease in accessing life-changing, sometimes lifesaving, medical treatments they otherwise would not be able to afford,” commented Krista Zodet, HealthWell Foundation President. “Thank you to our dedicated donors for recognizing this critical need and for helping us serve this patient community.”
To determine eligibility and apply for financial assistance, visit HealthWell’s Sickle Cell Disease Fund page. To learn how you can support this or other HealthWell programs, visit HealthWellFoundation.org
About the HealthWell Foundation
A nationally recognized, independent non-profit organization founded in 2003, the HealthWell Foundation has served as a safety net across over 70 disease areas for more than 500,000 underinsured patients. Since its inception, HealthWell has provided over $1.6 billion in grant support to access life-changing medical treatments patients otherwise would not be able to afford. HealthWell provides financial assistance to adults and children facing medical hardship resulting from gaps in their insurance that cause out-of-pocket medical expenses to escalate rapidly. HealthWell assists with the treatment-related cost-sharing obligations of these patients. HealthWell ranked 33rd on the 2019 Forbes list of the 100 Largest U.S. Charities and was recognized for its 100 percent fundraising efficiency. For more information, visit www.HealthWellFoundation.org.
 
About SCDAA
SCDAA’s mission is: To advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. Visit www.sicklecelldisease.org.
About SCD
SCD, an inherited blood disease, causes red blood cells to have a sickle shape. Because of their stiffness and unusual form, blood flow is blocked to different tissues, ultimately damaging them. These sickle-shaped red blood cells contain an abnormal type of hemoglobin, hemoglobin S; normal red blood cells have hemoglobin A. Hemoglobin is important because it helps carry oxygen throughout the body. There is currently no universal cure for SCD.
 
CONTACT:
Jacqueline Burrell
Director of Communications
JBurrell@sicklecelldisease.org
 

ASH and FDA Unveil New Recommendations to Guide Clinical Development of Sickle Cell Disease Therapies

Joint FDA/ASH Led Initiative Highlights Importance of Using Patient Reported Outcomes and Biomarkers in Clinical Trials to Advance SCD Therapies
(WASHINGTON, DC, Dec. 6, 2019) — The American Society of Hematology (ASH) today released the most comprehensive set of recommendations to date aimed at establishing uniformity and global standards for clinical trial endpoints used to evaluate new therapies for sickle cell disease (SCD). The new recommendations – published in two companion papers in the current issue of Blood Advances – are the result of seven expert and patient led panels convened by ASH and the U.S. Food and Drug Administration (FDA) to improve the design of clinical trials for new SCD therapies, including promoting broader use of patient reported outcomes and biomarkers as clinical endpoints.
Sickle cell disease is the most common inherited red blood cell disorder in the United States, and it affects millions of people worldwide. In people living with SCD, the red blood cells, which are normally round, become crescent or sickle-shaped which contributes to the vaso-occlusive crises these patients experience. People with SCD suffer from an array of physical complications, including acute pain crises, joint and organ damage, impaired cognitive function, and a reduced life expectancy. In addition to the immense physical burden they must endure, people with SCD are often stigmatized due to a poor understanding among healthcare professionals and the general public of the life-limiting effects of the disease. Supporting SCD research and access to care efforts for all people living with the disease is a chief priority for ASH.
While the molecular basis of SCD has been well understood for decades, there are currently only four FDA approved treatments for this debilitating condition. Bone marrow transplant is a cure for some individuals with SCD, but it is not an option for everyone. Although two new treatments for SCD have recently been approved, and many others – including potentially curative gene therapies – are in development, medical experts and FDA officials agree there is a need for uniformity around clinical trial endpoints to ensure these new therapies deliver a meaningful benefit from the patient’s perspective. “There are a number of investigational drugs in development that target different manifestations of SCD,” said Julie Panepinto, MD, MSPH, FAAP, Professor of pediatric hematology, Medical College of Wisconsin/Children’s Wisconsin, co-chair of ASH’s Guideline Oversight Committee, and co-chair of the workshop. “However, there are no clear standardized endpoints for evaluating the effect of therapies on clinical outcomes and patient well-being.”
Dr. Panepinto said that amid the burgeoning effort to develop curative SCD therapies, clinical research should incorporate endpoints that are not only measurable, but also relevant and directly beneficial to the patient based on their preferences and experience.
“What’s happening in SCD is really exciting and many of us feel we are on the cusp of identifying multiple disease-modifying therapies,” Dr. Panepinto said. “The field is exploding, so we want to be sure we are measuring relevant endpoints for researchers, clinicians, and patients because that helps us advance the field and get new therapies approved.”
Ann Farrell, MD, Director of the FDA’s Division of Hematology Products and co-chair of the workshop, points out that the scientific understanding and the treatment of SCD have evolved to a point that the endpoints used to evaluate earlier SCD treatments are now inadequate.
“These changes have greatly impacted the discussion the Agency is having with the pharmaceutical industry as new clinical trials are designed,” said Dr. Farrell. “We need endpoints that better reflect the patient experience of their disease in the current healthcare system.”
To address the global burden of SCD, ASH and the FDA convened seven panels of clinicians, investigators, and people with SCD in a two-day workshop to bring uniformity and standards to existing endpoints, identify gaps, and propose development of new endpoints as a focus for future research. Led by Drs. Panepinto and Farrell, the panels conducted extensive literature reviews, assessed available evidence, and used expert judgement to identify which existing endpoints can be incorporated into SCD clinical trials and what additional data are needed. The panels focused on the following areas:
· Patient reported outcomes (PROs) – the panel suggested that future trials should measure the impact in three key domains: crisis and non-crisis pain; affect (including emotional impact, sleep quality and fatigue), and function (social, physical and cognitive),
· Pain (non-PROs) – this panel recommended measuring healthcare utilization, analgesic use, and physical function as a complementary measure to PROs on pain,
· The brain – the panel reviewed and identified diagnostic modalities to assess neurological risk, document stroke, and measure cognition and educational attainment,
· End-organ considerations – the panel stressed the need to use biomarkers and endpoints that capture the progression of renal and cardiopulmonary disease,
· Biomarkers – this panel overlapped with other panels addressing various disease manifestations, highlighting the importance of developing and validating a broader array of biomarkers that can measure response to therapy,
· Measurements of cure – given this is a relatively new area of research, the panel identified the need to develop appropriate biomarkers to evaluate the effect of curative therapies, and to capture and share these data in a central repository to help advance the scientific understanding and broader use of these treatments,
· Care in low-resource settings – this panel noted an opportunity to accelerate clinical trials in regions with a high prevalence of SCD, such as sub-Saharan Africa. They also recommended capturing data on early childhood, peri-operative and pregnancy-related mortality, as well as PROs from children and their caregivers relating to growth and development.
The published recommendations represent the most comprehensive review of science to date in the treatment of SCD and will be a valuable reference for academic researchers seeking funding for scientific studies and pharmaceutical companies looking to identify endpoints for specific clinical trials.
“The papers document the wide-ranging discussions held by researchers, patients, caregivers, international experts, pharmaceutical industry and government to understand where we are in terms of defining clinical trial endpoints for current use and those for future development that will serve patients best,” said Dr. Farrell. She also noted the initiative identified several opportunities to incorporate the patient voice in clinical practice to help better understand how effective current medical treatments are.
The workshop was modeled after an earlier ASH/FDA initiative that focused on leukemia and myeloma, and was generously supported solely by the philanthropic support from numerous individual donors who contributed to the ASH Foundation’s Sickle Cell Disease Initiative Fund and by the Doris Duke Charitable Foundation.
In 2016, ASH launched a multifaceted initiative to address the burden of disease both in the United States and globally. For this initiative, ASH has developed clinical guidelines for SCD management and care, expanded education and training efforts, advocated with policymakers to enhance and expand federal SCD programs, and founded the Sickle Cell Disease Coalition. In addition to these efforts, the ASH Research Collaborative (ASH RC) SCD Clinical Trials Network was developed with the mission to improve outcomes for individuals with SCD by expediting SCD therapy development and facilitating innovation in clinical trial research. It provides the infrastructure for identifying patient cohorts for trials, matching trial sponsors with sites, facilitating recruitment of eligible patients, and ensuring optimally designed trials and an efficient, coordinated approach. Through patient engagement and optimized clinical trial execution, the Clinical Trials Network is helping to bring new and more effective therapies to individuals with SCD.
Papers in Blood Advances:
· End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain
· End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource setting
The American Society of Hematology (ASH) (www.hematology.org) is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, and Blood Advances (www.bloodadvances.org), an online, peer-reviewed open-access journal. 

GBT’s Oxbryta™ (voxelotor) tablets is approved for the treatment of sickle cell disease

Oxbryta™ (voxelotor) tablets Now Approved

On behalf of GBT, we are happy to share that Oxbryta (pronounced ox-brye-ta) is now approved by the U.S. Food and Drug Administration (FDA). Oxbryta is a prescription medicine used for the treatment of sickle cell disease in adults and children 12 years of age and older.1 It is not known if Oxbryta is safe and effective in children below 12 years of age.1
This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).2
This approval has been the result of years of collaboration with the community, and we are so grateful for your role in helping GBT get this to patients quickly!
1) Oxbryta is the brand name for voxelotor. This approval represents a historic one, as Oxbryta is the first sickle cell disease drug approved under both the Breakthrough Therapy and Accelerated Approval designations by the FDA.
· Voxelotor is a hemoglobin S (HbS) polymerization inhibitor.2
· Nonclinical studies suggest that voxelotor may inhibit red blood cell (RBC) sickling, improve RBC deformability, and reduce whole blood viscosity.2
· It is a prescription medicine (tablet) taken by mouth once daily, every day.1
2) Oxbryta should not be taken if a patient has had an allergic reaction to voxelotor or any of the ingredients in Oxbryta. Oxbryta can cause side effects including: headache, diarrhea, stomach (abdominal) pain, nausea, tiredness, rash and fever.1
Patient Support: GBT Source Solutions™
As part of GBT’s commitment to supporting access to care for patients, we have launched GBT Source Solutions, a resource center for patients who have been prescribed Oxbryta by their healthcare provider. GBT Source Solutions will provide support by:
· Reviewing insurance coverage options and explaining benefits.
· Coordinating shipment of Oxbryta and explaining Specialty Pharmacy benefits.
· Helping to pay for treatment with financial and co-pay assistance for eligible patients.
· Helping to stay on treatment with a nurse support team. The nurse support team is there to support product adherence, and not to replace a patient’s treatment plan. They do not provide medical advice or case management services.
Your role
Your organization plays a critical role in supporting patients, and we are very thankful for everything you do for people living with SCD. Your collaboration is critical to helping inform patients about Oxbryta and GBT’s patient support services for patients prescribed Oxbryta.
If a patient is interested in learning more about Oxbryta, they should consult their healthcare professional or visit www.Oxbryta.com.
We have attached in this email several resources. You may share these with patients, caregivers, and other members of your community.
… Oxbryta fact sheet
… Oxbryta patient education brochure
… GBT Source Solutions brochure
Please feel free to reach out with any questions. We look forward to continuing our collaboration to help improve the lives of SCD patients and provide hope to our community.
Warmest regards,
Jung
INDICATION AND IMPORTANT SAFETY INFORMATION
INDICATION
What is OXBRYTA?
OXBRYTA is a prescription medicine used for the treatment of sickle cell disease in adults and children 12 years of age and older.
It is not known if OXBRYTA is safe and effective in children below 12 years of age.
This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
IMPORTANT SAFETY INFORMATION
Do not take OXBRYTA if you have had an allergic reaction to voxelotor or any of the ingredients in OXBRYTA. See the end of the patient leaflet for a list of the ingredients in OXBRYTA.
If you are receiving exchange transfusions, talk to your healthcare provider about possible difficulties with the interpretation of certain blood tests when taking OXBRYTA.
Before taking OXBRYTA, tell your healthcare provider about all of your medical conditions, including if you:
· have liver problems
· are pregnant or plan to become pregnant. It is not known if OXBRYTA can harm your unborn baby.
· are breastfeeding or plan to breastfeed. It is not known if OXBRYTA can pass into your breastmilk and if it can harm your baby. Do not breastfeed during treatment with OXBRYTA and for at least 2 weeks after the last dose.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Some medicines may affect how OXBRYTA works. OXBRYTA may also affect how other medicines work.
What are the possible side effects of OXBRYTA?
OXBRYTA can cause serious side effects, including:
Serious allergic reactions. Tell your healthcare provider or get emergency medical help right away if you get:
· rash
· hives
· shortness of breath
· swelling of the face
The most common side effects of OXBRYTA include:
· headache
· diarrhea
· stomach (abdominal) pain
· nausea
· tiredness
· rash
· fever
These are not all the possible side effects of OXBRYTA.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
You may also report side effects to Global Blood Therapeutics at 1-833-428-4968 (1-833-GBT-4YOU).
Keep OXBRYTA and all medicines out of the reach of children.
1 Patient Information Leaflet 11 2019
2 USPI 11 2019  

CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data

Nov 19, 2019

CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001® for Severe Hemoglobinopathies

-Two patients treated with CTX001 successfully engrafted and demonstrated an initial safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant-
-Beta thalassemia: Patient is transfusion independent with total hemoglobin level of 11.9 g/dL and 10.1 g/dL fetal hemoglobin at nine months after CTX001 infusion-
-Sickle cell disease: Patient is free of vaso-occlusive crises with total hemoglobin level of 11.3 g/dL and 46.6% fetal hemoglobin at four months after CTX001 infusion-
-CRISPR Therapeutics will host a conference call today at 8:00 a.m. ET to review these data-
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 19, 2019 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. One patient with transfusion-dependent beta thalassemia (TDT) received CTX001 in the first quarter of 2019 and data for this patient reflect nine months of safety and efficacy follow-up. One patient with severe sickle cell disease (SCD) received CTX001 in mid-2019 and data for this patient reflect four months of safety and efficacy follow-up. These studies are ongoing and patients will be followed for approximately two years following infusion. Several additional patients have been enrolled and have had drug product manufactured across the two studies.
Transfusion-Dependent Beta Thalassemia
The patient with TDT has the β0/IVS-I-110 genotype and required 16.5 transfusions per year (annualized rate during the two years prior to consenting for the study) before enrolling in the clinical study. The patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. Two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. At nine months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells (erythrocytes expressing fetal hemoglobin).
Sickle Cell Disease
The patient with SCD experienced seven vaso-occlusive crises (VOCs) per year (annualized rate during the two years prior to consenting for the study) before enrolling in the clinical study. The patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. Three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis, and abdominal pain, all of which resolved. At four months after CTX001 infusion, the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells (erythrocytes expressing fetal hemoglobin).
“We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate, CTX001,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention. We continue to enroll these studies as we drive forward to develop CRISPR/Cas9 therapies as a new class of transformative medicines to treat serious diseases.”
“The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia,” said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. “While the data are exciting, we are still in the early phase of this clinical program. We look forward to continuing to work with physicians, patients, caregivers and families over the coming months and years to bring forward the best possible therapy for these two serious diseases and to continue to accelerate our gene-editing programs for other serious diseases such as Duchenne muscular dystrophy and myotonic dystrophy type 1.”
About the Phase 1/2 Study in Transfusion-Dependent Beta Thalassemia
The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study. Enrollment is ongoing at six clinical trial sites in the United States, Canada and Europe.
About the Phase 1/2 Study in Sickle Cell Disease
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study. Enrollment is ongoing at 12 clinical trial sites in the United States, Canada and Europe.
About the Gene–Editing Process in These Trials
Patients who enroll in these studies will have hematopoietic stem and progenitor cells collected from peripheral blood. The patient’s cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease.
CRISPR Therapeutics Conference Call and Webcast
CRISPR Therapeutics will host a conference call and webcast today at 8:00 a.m. ET. The webcast and presentation will be made available on the CRISPR Therapeutics website at https://crisprtx.gcs-web.com/events in the Investors section under Events and Presentations. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.
Dial-In Information
Live (U.S. / Canada): (800) 895-3361
Live (International): (785) 424-1062
Conference ID: 87198237
About CTX001
CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.
CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.
About the CRISPR-Vertex Collaboration
CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.
About CRISPR Therapeutics
CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc.CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit www.crisprtx.com.
CRISPR Therapeutics Forward-Looking Statement
This press release may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics’ expectations about any or all of the following: (i) the safety, efficacy and clinical progress of CRISPR Therapeutics’ CTX001 clinical program; (ii) the status and scope of ongoing and potential future clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof and the timing of commencement of clinical trials), development timelines and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators; (iii) the number of patients that will be evaluated, the anticipated date by which enrollment will be completed and the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; v(iv) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties; (v) the sufficiency of CRISPR Therapeutics’ cash resources; and (vi) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words “believes,” “anticipates,” “plans,” “expects” and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial (including CTX001) not to be indicative of final trial results; the risk that the initial data from a limited number of patients (as is the case with CTX001 at this time) may not be indicative of results from the full planned study population; the outcomes for each CRISPR Therapeutics’ planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics’ internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics’ product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics’ product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics’ technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading “Risk Factors” in CRISPR Therapeutics’ most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC’s website at www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency, and APOL1-mediated kidney disease. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex’s global headquarters is now located in Boston’sInnovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry’s top places to work, including 10 consecutive years on Science magazine’s Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
(VRTX-GEN)
Vertex Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding the status of, and expectations with respect to, the CTX001 clinical development program. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release, and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include that the development of CTX001 may not proceed due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex’s annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company’s website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
CRISPR Therapeutics Investor Contact:
Susan Kim, +1 617-307-7503
susan.kim@crisprtx.com
CRISPR Therapeutics Media Contact:
Jennifer Paganelli
WCG on behalf of CRISPR
+1 347-658-8290
jpaganelli@wcgworld.com
Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1 617-341-6108
or
Zach Barber, +1 617-341-6470
or
Leah Gibson, +1 617-961-1507
Media: mediainfo@vrtx.com
or
North America:
Heather Nichols, +1 617-341-6992
Heather_Nichols@vrtx.com

 

Novartis Adakveo approved by FDA for Sickle Cell Disease


New Novartis medicine Adakveo® (crizanlizumab-tmca) approved by FDA to reduce frequency of pain crises in individuals living with sickle cell disease

  • Sickle cell pain crises are unpredictable, severe events associated with life-threatening complications1
  • Adakveo reduced the annual rate of sickle cell pain crises by 45% compared to placebo (1.63 vs 2.98) and the annual rate of days hospitalized (4 vs 6.87) in a 52-week study2
  • Approximately 100,000 people in the United States, most of whom are of African descent, have sickle cell disease3
  • Approval comes approximately two months ahead of FDA’s priority review action date, allowing Adakveo to be available to patients more quickly

East Hanover, NJ, November 15, 2019 – Novartis announced today that the US Food and Drug Administration (FDA) approved Adakveo® (crizanlizumab-tmca), previously known as SEG101, to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell disease.4 Adakveo represents the first FDA-approved medicine in sickle cell disease that binds to P-selectin – a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.5,6 The medicine is expected to be available to patients in the coming weeks.
The FDA’s decision to approve Adakveo 5 mg/kg is based on results of the 52-week, randomized, placebo-controlled SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo (P=.010), which is equivalent to a 45% reduction. Reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype and/or hydroxyurea use.2,4
“We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way,” said Kenneth Ataga, MD, Director, Center for Sickle Cell Disease, University of Tennessee Health Science Center at Memphis, and Principal Investigator of the SUSTAIN trial. “The approval of crizanlizumab is an important advancement for people living with this very difficult condition.”
Additional results from the SUSTAIN study include:4

  • A decrease in the median annual rate of days hospitalized to 4 vs 6.87 days when compared with placebo (a 42% reduction)
  • Thirty-six percent of patients treated with Adakveo did not experience a VOC, compared to 17% of placebo-treated patients
  • The median time to first VOC was 4.1 for Adakveo vs 1.4 months for placebo

The most common adverse reactions (incidence > 10%) were nausea (18%), arthralgia (18%), back pain (15%) and pyrexia (11%).4
“The approval of Adakveo marks a new era in the treatment of sickle cell disease, a genetic condition that places an extraordinary burden of unpredictable pain crises on patients and their families,” said Susanne Schaffert, PhD, President, Novartis Oncology. “The stories we have heard from patients about their sickle cell pain crises are devastating. We are pleased to help reimagine medicine together with the sickle cell community and offer new hope for fewer VOCs.”
Considered the clinical hallmark of the disease, sickle cell pain crises are triggered, in part, by multicellular interactions that form clusters of cells, which can block or reduce the blood flow to organs.1,7 Sickle cell pain crises can be frequent and sudden, and are associated with an increased risk of life-threatening complications.1 They also are the main reason why individuals living with sickle cell disease go to the emergency room and are admitted to the hospital.7
“Patients with sickle cell disease often face unique challenges, and have long suffered silently through unimaginable pain crises,” said Beverley Francis-Gibson, President and CEO of the Sickle Cell Disease Association of America. “We are excited to have a new medicine that may help many of the thousands of people living with sickle cell disease by reducing the frequency of these potentially dangerous and painful episodes.”
About Sickle Cell Disease
Sickle cell disease is a complex and debilitating genetic blood disorder that goes beyond sickle-shaped red blood cells. The disease is associated with chronic inflammation, causing higher levels of cell adhesion proteins, including P-selectin, which make both the blood vessels and certain blood cells stickier and prone to multicellular interactions, or clusters, in the bloodstream. This environment can lead to the acute episodes of pain known as sickle cell pain crises, or VOCs, as well as life-threatening complications.1,7,8 VOCs are the main reason why individuals living with sickle cell disease seek medical care in hospitals,7 leading to approximately 200,000 ER visits in the US every year.9,10
Approximately 100,000 people in the US have sickle cell disease.3 People of African ancestry make up 90% of the population with sickle cell disease in the US. However, sickle cell disease is also prevalent among people of Hispanic, South Asian, Southern European, and Middle Eastern ancestry. Sickle cell disease occurs in about 1 in 365 and 1 in 16,300 African-American and Hispanic-American births, respectively.3
About Adakveo
Adakveo® (crizanlizumab-tmca) – previously known as SEG101 – is indicated to reduce the frequency of VOCs, or pain crises, in adults and pediatric patients aged 16 years and older with sickle cell disease. It is the first and only targeted biologic that works by binding to P-selectin, a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion in sickle cell disease.
By binding to P-selectin on the surface of the activated endothelium and platelets, Adakveo blocks interactions between endothelial cells, platelets, red blood cells, and leukocytes.4
About SUSTAIN
SUSTAIN is a randomized, multicenter, placebo-controlled, double-blind study. A total of 198 patients with any genotype of sickle cell disease (HbSS, HbSC, HbS/beta0-thalassemia, HbS/beta+-thalassemia, and others) and a history of 2-10 VOCs in the previous 12 months were eligible for inclusion. Patients were randomized 1:1:1 to Adakveo 5 mg/kg (N = 67), Adakveo 2.5 mg/kg (N = 66), or placebo (N = 65) administered over a period of 30 minutes by intravenous infusion on Week 0, Week 2, and every 4 weeks thereafter, for a treatment duration of 52 weeks.
The primary efficacy outcome was the annual rate of VOCs leading to a healthcare visit. A VOC leading to a healthcare visit was defined as an acute episode of pain with no cause other than a vaso-occlusive event that required a medical facility visit and treatment with oral or parenteral opioids, or parenteral NSAIDs. Acute chest syndrome, hepatic sequestration, splenic sequestration, and priapism (requiring a visit to a medical facility) were also considered VOCs. Key secondary and other efficacy endpoints include annual rate of days hospitalized, time to first VOC leading to healthcare visit, and number of patients that did not experience a VOC.
Patient Access and Support
Novartis is committed to helping ensure that our medicines are accessible to as many patients as possible. With the approval of Adakveo in the United States, we now offer resources and support to address a range of needs. Adakveo Support at PANO (Patient Assistance Now Oncology) is a support center staffed by insurance specialists and case managers who can help eligible patients start and stay on treatment. Dedicated support specialists are available to help direct callers to services that best fit their needs. Patients or providers can call 800-282-7630 or visit Patient.NovartisOncology.com or HCP.Novartis.com/Access to learn more about eligibility and to enroll.
Novartis Commitment to Sickle Cell Disease in Africa
Sickle cell is a global disease and is most widespread in sub-Saharan Africa. Unfortunately, we can see a clear disparity when comparing Africa with other parts of the world, where sickle cell is often managed as a chronic disease. Building on years of engagement in Africa, working to reduce the impact of malaria and other conditions, Novartis is taking steps to help address the needs of sickle cell patients as well, beginning in Ghana. Our partnership with the Ghana Ministry of Health, the Ghana Health Service, and the Sickle Cell Foundation of Ghana aims to improve the diagnosis and treatment of people with sickle cell disease through a comprehensive approach to screening and diagnosis, treatment and disease management, training and education, and elevating basic and clinical research and scientific capabilities. These activities include facilitating access to high-quality hydroxyurea and other basic medicines to enhance the standard of care.
To date, Novartis has delivered more than 20,000 hydroxyurea treatments to Ghana, with plans to deliver a total of 60,000 treatments by the end of the year. In addition, Novartis is developing a child-friendly formulation of hydroxyurea and is committed to implementing two clinical trials with crizanlizumab in Ghana and Kenya – an important step to bringing this innovative medicine to patients. Crizanlizumab trials in Africa are expected to start in 2020.
Indication
Adakveo® (crizanlizumab-tmca) is used in people 16 years of age and older, who have sickle cell disease, to help reduce how often certain episodes of pain (crises) happen. It is not known if Adakveo is safe and effective in children under 16 years of age.
Important Safety Information
Adakveo may cause serious side effects, including infusion reactions. Infusion reactions may happen within 24 hours of receiving an infusion of Adakveo. Patients should tell their health care provider right away if they get any of the following signs and symptoms of an infusion reaction such as fever, chills or shivering, nausea, vomiting, tiredness, dizziness, sweating, hives, itching, or shortness of breath or wheezing. Health care providers may monitor their patients for signs and symptoms of infusion reactions.
Adakveo may interfere with automated platelet counts (platelet clumping). Patients should tell their health care provider that they are receiving Adakveo before having any blood tests. Health care providers should run blood samples as soon as possible or use tubes containing citrate.
Before receiving Adakveo, patients should tell their health care provider if they are pregnant or plan to become pregnant. It is not known if Adakveo may harm an unborn baby.
The most common side effects (incidence ≥10%) include nausea, back pain, joint pain, and fever.
Please see full Prescribing Information for Adakveo at https://sicklecelldisease.org/wp-content/uploads/2024/01/adakveo.pdf.
Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political and economic conditions; safety, quality or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
About Novartis
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. Novartis Pharmaceuticals Corporation, a US affiliate of Novartis, is located in East Hanover, NJ. Find out more at www.novartis.com.
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References

  1. Steinberg M. Management of sickle cell disease. N Engl J Med. 1999;340(13):1021-1030.
  2. Ataga KI, Kutlar A, Kanter J et al. Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med. 2017;376(5):429-439.
  3. American Society of Hematology. State of sickle cell disease 2016 report. http://www.scdcoalition.org/pdfs/ASH%20State%20of%20Sickle%20Cell%20Disease%202016%20Report.pdf. Accessed October 24, 2019.
  4. Adakveo (crizanlizumab) prescribing information. East Hanover, New Jersey, USA. Novartis Pharmaceuticals Corporation; November 2019.
  5. Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010;376(9757):2018-2031.
  6. Lawrence MB, Springer TA. Leukocytes roll on a selectin at physiologic flow rates: distinction from and prerequisite for adhesion through integrins. Cell. 1991;65(5):859-873.
  7. Gutsaeva D, Parkerson J, Yerigenahally S, et al. Inhibition of cell adhesion by anti–P-selectin aptamer: a new potential therapeutic agent for sickle cell disease. Blood. 2011;117(2):727-735.
  8. Sparkenbaugh E, Pawlinski R. Interplay between coagulation and vascular inflammation in sickle cell disease. Br J Haematol. 2013;162(1):1-22.
  9. Ballas SK, Gupta K, Adams-Graves P. Sickle cell pain: a critical reappraisal. Blood. 2012;120(18):3647-3656.
  10. Yusuf HR, Atrash HK, Grosse SD, Parker CS, Grant AM. Emergency department visits made by patients with sickle cell disease: a descriptive study, 1999-2007.Am J Prev Med. 2010;38(Suppl):S536-S541.

Novartis Global External Communications
E-mail: media.relations@novartis.com