News

MARAC Statement: Response to Oxbryta® Update August 6, 2026 – The Medical Research and Advisory Committee (MARAC) of the Sickle Cell Disease Association of America, Inc., (SCDAA) is saddened to hear the news that Oxbryta® (voxelotor) will not be made available for the treatment of sickle cell disease. Based on recent engagement with the Food and Drug Administration (FDA), Pfizer was informed that there is not a viable path to make Oxbryta available again in the U.S. We understand that this news is disappointing, especially given the significant unmet needs for people living with sickle cell. In their statement, Pfizer confirmed its continued commitment to the sickle cell community and stated that they will be concentrating their ongoing research efforts on osivelotor, a sickle hemoglobin (HbS) polymerization inhibitor being investigated as a potential treatment for sickle cell disease (SCD). Pfizer will also continue to share Oxbryta data as it is published and has agreed to engage with MARAC in the near future to discuss these developments. MARAC’s sickle cell healthcare providers and researchers will continue to represent the sickle cell community in conversations with Pfizer and other industry groups and will help keep the sickle cell community informed of developments. Although progress has been made in the treatment of sickle cell disease, there is still a limited number of therapies available for individuals suffering from the disease. MARAC encourages the pharmaceutical industry and the FDA to work with the sickle cell community to continue to look for therapies that not only address the clinical outcomes that are often evaluated in sickle cell studies but to also explore outcome measures and endpoints that address patient-reported outcomes that impact quality of life. MARAC looks forward to continuing to partner with the pharmaceutical industry, government and the sickle cell community to find treatments for the sickle cell population. Download the statement here. The post MARAC Statement: Response to Oxbryta® Update first appeared on SCDAA.

As we get closer to the SCDAA Annual Convention we would like to recognize the amazing work performed by community health workers (CHWs) with the SCDAA CHW P.O.W.E.R. Award. This award recognizes exemplary performance by individuals 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. The post Nominate a CHW for the 2026 CHW P.O.W.E.R Award first appeared on SCDAA.

The Medical Research and Advisory Committee (MARAC) of the Sickle Cell Disease Association of America, Inc., (SCDAA) is excited about the Food and Drug Administration’s (FDA) approval of Casgevy (exagamglogene autotemcel) for sickle cell patients aged 2 years and older. We are encouraged to learn they are “committed to prioritizing and speeding up the review of products that address critical U.S. health priorities,” including sickle cell disease (SCD). The post MARAC Statement: FDA Approval of First Gene Therapy for Children Ages 2+ With SCD first appeared on SCDAA.

The success of any organization, for profit or not for profit, is measured by its leadership. As organizational leaders, we value the commitment, vision and passion our board members bring. The board of directors for Sickle Cell Disease Association of America, Inc. (SCDAA) looks to those who serve – or have served – so passionately, to nominate qualified individuals to the board of directors for SCDAA. The post SCDAA Seeks New Board Members first appeared on SCDAA.

The Sickle Cell Disease Association of America Inc. urges the U.S. Department of Health and Human Services (HHS) and its Health Resources and Services Administration (HRSA) to issue the funding opportunity for the Sickle Cell Disease Newborn Screening Follow-Up Program to ensure the next five-year grant cycle, which is set to start Sept. 1, proceeds without delay. The post SCDAA Urges Renewal of Critical Sickle Cell Program first appeared on SCDAA.

The Sickle Cell Disease Association of America, Inc. (SCDAA) Medical and Research Advisory Committee (MARAC) is saddened to hear the news that Fulcrum Therapeutics is discontinuing development of its pociredir program for the treatment of sickle cell disease. The post MARAC Statement on Pociredir first appeared on SCDAA.

From May 6-7, 24 representatives from 13 SCDAA members organizations across 11 states traveled to Washington, D.C., for SCDAA’s Advocacy Days. Attendees participated in a briefing on May 6, which included information about how to have a successful Congressional meeting, an overview of the issues and asks and a visit from Representative Glenn Ivey (D-MD-04), one of the new co-chairs of the recently relaunched Congressional Sickle Cell Disease Caucus. On May 7, advocates took to Capitol Hill and met with 34 offices to ask Members of Congress to support the sickle cell disease community. The post SCD Advocacy Update June 2026 first appeared on SCDAA.

The National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH), in partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA), will host Research That Heals: Partnering to Transform SCD Care on June 25-26, 2026. This 1.5-day hybrid (in-person and virtual) community forum will be a crucial platform for engaging directly with the sickle cell disease (SCD) community, including individuals with SCD and their caregivers, healthcare providers and community-based organizations. During the forum, participants will discuss and develop solutions to enhance care and improve the quality of life for children and adults living with sickle cell disease. The post SCDAA and NHLBI To Host Event first appeared on SCDAA.

The 2026 National Abstract Convention is now open! If you are a researcher, community-based member organization, physician, nurse, social worker or someone working on behalf of people living with sickle cell disease and their families, the Sickle Cell Disease Association of America, Inc. would like to highlight your work at the 54th Annual National Convention, October 15-17, 2026, in-person. The post 2026 National Abstract Competition first appeared on SCDAA.

In an announcement, Novo Nordisk shared that Etavopivat is the first in a new class of drugs to meet both co-primary endpoints in the phase 3 HIBISCUS trial, substantially reducing vaso-occlusive crisis events and improving hemoglobin response in sickle cell disease. The post Novo Nordisk Announces Promising Topline HIBISCUS Study Results first appeared on SCDAA.