August 15, 2025 – In a statement issued today, Pfizer announced the results of their Phase 3 THRIVE-131 study evaluating inclacumab. Although inclacumab was generally well tolerated in THRIVE-131, the study results concluded that inclacumab “did not meet its primary endpoint of significant reduction in the rate of vaso-occlusive crises (VOCs) in participants receiving inclacumab versus placebo every 12 weeks.” The Medical and Research Advisory Committee (MARAC) of the Sickle Cell Disease Association of America, Inc. (SCDAA) would like to express its disappointment in hearing this news. While we recognize that not all therapies will be approved, this report does present a setback to the sickle cell disease community. It underscores the need for further research and clinical trials to find a universal cure for this rare disease.

Sickle cell disease (SCD) is a rare condition that affects over 100,000 Americans and millions across the world. Progress has been made in the treatment of the disease since the approval of hydroxyurea 1997 but it has been slow and far and few between. In addition to hydroxyurea, only two other medications are currently available to treat sickle cell patients: L- glutamine and crizanlizumab. These disease modifying therapies have successfully addressed such major complications as acute pain crisis and acute chest syndrome, among others. Still individuals continue to report issues such as fatigue, chronic pain and other symptoms that result in poor quality of life. Recently approved cell and gene therapies are available and are potentially curative but are costly and require a significant time commitment for the transplant procedure.

MARAC recognizes that while the Pfizer study did not achieve the anticipated outcomes, we applaud those individuals who participated in this and other in clinical trials. Had the therapy achieved its primary clinical trial endpoint, it would have been promising. MARAC encourages the pharmaceutical industry and the Food and Drug Administration (FDA) to work with the sickle cell community to continue to search for therapies that not only address the clinical outcomes often evaluated in sickle cell studies but also explore outcome measures and endpoints that address patient-reported outcomes that impact quality of life.

SCDAA has also joined with a community of sickle cell disease advocacy organizations with this response.

Exciting News: the Maryland Department of Health has officially accredited SCDAA’s Community Health Worker Training! This accolade will allow our program to have an even bigger impact in the state. Students who have successfully completed our training program can now apply for CHW certification with the Maryland Department of Health. Congrats to our CHW team on this accomplishment!

Each year during the SCDAA Annual National Convention, we recognize the amazing work performed by community health workers (CHWs) with the SCDAA P.O.W.E.R. CHW Award. The CHW Award honors exemplary performance by individuals who have been identified by their supervisors and peers as having consistently excelled in their positions and demonstrated integrity and a strong commitment to the sickle cell disease (SCD) community and values of the community health worker profession.

If you would like to nominate a CHW for the award, please fill out the below document and submit to the SCDAA Senior Community Impact & Engagement Manager Kevin Amado at kamado@sicklecelldisease.org. The deadline for submission is Friday, August 29.

• Download a Word Doc
• Download a PDF file

On July 15, 2025, the Centers for Medicare & Medicaid Services (CMS) announced expansions to the Cell and Gene Therapy (CGT) Access Model.

“The Sickle Cell Disease Association of America, Inc., is excited by the news that CMS has reached agreements with drug manufacturers to provide gene therapies to treat sickle cell disease to Medicaid recipients and that 33 states, plus the District of Columbia and Puerto Rico, have joined a program that ties Medicaid payments to patient outcomes,” says Dr. Edward Donnell Ivy, SCDAA chief medical officer. “For a long time, the only disease-modifying therapy for sickle cell disease was hydroxyurea, which doesn’t work for many sickle cell patients and leaves the community with limited options for care. The new gene therapies that were approved by the FDA in 2023 will allow individuals the opportunity for transformative therapy with potential cure. However, access to this new therapy might be limited by the cost of the treatment, so it is important for CMS and other stakeholders to get involved in ensuring access to this therapy, particularly since it has been demonstrated that a large portion of the sickle cell population relies on Medicaid for access to care.”

To learn more, click here to read the CMS statement.

On July 4, President Trump signed the One Big Beautiful Bill Act into law. SCDAA shares the concerns of many in our SCD community regarding the impact that this act will have on the Medicaid program, access to care and health care overall for sickle cell warriors. We are in the process of carefully analyzing the bill to fully understand its effects and develop guidance for warriors, providers, caregivers and families moving forward. We will keep you updated in the next few weeks with more information and proposed next steps.

SCDAA will continue to advocate on both the federal and state levels as provisions of the law are implemented. Thanks to the many of you who engaged in advocacy on the bill. We are a resilient community and our work must continue.