ASH Statement on FDA’s Approval of New Sickle Cell Disease Gene Therapy

New class of drugs represent progress, but full comprehensive care is needed

(WASHINGTON, December 8, 2023) – The U.S. Food and Drug Administration (FDA) approved Vertex and Bluebird's gene editing therapies for sickle cell disease (SCD) today, marking the first approval of a new class of drugs to cure sickle cell disease.

SCD is the most common inherited red blood cell disorder in the United States, affecting an estimated 100,000 people. According to the Centers for Disease Control and Prevention, SCD affects one out of every 365 Black or African American births and one out of every 16,300 Hispanic American births.

ASH President Dr. Robert A. Brodsky, of Johns Hopkins University School of Medicine, issued the following statement:

“The approval of the first gene therapies for SCD represent a tremendous step forward for the SCD community, which has been historically overlooked and underfunded. While these new gene therapies are potentially life-changing for individuals living with SCD, they must be accessible to be effective.

Despite the recent progress in expanding SCD therapies, current treatments and treatment models do not adequately address the many barriers to care people living with SCD face, including access to providers that understand their condition, hydroxyurea for symptom management, pain medication during pain crises, and specialized and high-quality care.

ASH remains committed to improving the availability of innovative treatments for blood disorders such as gene therapies for SCD and providing resources to clinicians to help implement these evolving therapies. People with SCD need more – they need comprehensive care.”

Resources

The American Society of Hematology (ASH) (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’s flagship journal, Blood (bloodjournal.org) is the most cited peer-reviewed publication in the field, and Blood Advances (bloodadvances.org) is an open-access, online journal that publishes more peer-reviewed hematology research than any other academic journal worldwide. Two new journals will be joining the Blood Journals portfolio in 2024, Blood Neoplasia (bloodneoplasia.org) and Blood Vessels, Thrombosis & Hemostasis (bloodvth.org).

Contact:

Melissa McGue, American Society of Hematology
mmcgue@hematology.org, (202) 552-4927