Access, Affordability Concerns Follow FDA OK Of Sickle Cell Gene Therapy
The FDA authorized on Friday a pair of gene therapies for sickle cell disease, one of which is the first to gain such approval using the CRISPR gene-editing system. News outlets report on costs and paths to patients for the treatments.
The New York Times:
New Sickle Cell Therapies Will Be Out Of Reach Where They Are Needed Most
The Food and Drug Administration’s approval on Friday of two groundbreaking gene therapy treatments for sickle cell disease has brought a rare moment of hope and celebration to people with the agonizing blood disorder. But there is no clear path for the new therapies — one-time treatments so effective in clinical trials that they have been hailed as cures — to reach the countries where the vast majority of people with sickle cell live. Shortly after the approval their manufacturers announced sticker prices in the millions of dollars: $3.1 million for Lyfgenia, made by Bluebird Bio, and $2.2 million for Casgevy, made by Vertex Pharmaceuticals. *Robbins and Nolen, 12/8)
Axios:
New Sickle Cell Gene Therapies Pose Cost And Access Questions
The approvals of Casgevy and Lyfgenia mark a milestone in the treatment of a debilitating disease that affects an estimated 100,000 Americans, mostly of African descent, and 20 million people around the world. ... Until now, they've have had to turn to bone marrow transplants. But the new therapies edit genes directly in a patient's body and expand the kinds of diseases and conditions researchers can target to other blood disorders, certain cancers and infectious diseases. (Bettelheim, 12/9)
Stat:
Experts At ASH Cautiously Optimistic About Sickle Cell Gene Therapies
News of the Food and Drug Administration’s historic approval of the first gene therapies for sickle cell disease sparked discussion, debate, and, above all, measured optimism at this year’s meeting of the American Society of Hematology. (Wosen, 12/9)
WMFE:
New Gene-Editing Treatment For Sickle Cell Could Be Great News In Central Florida
The Food and Drug Administration on Friday approved a gene-editing therapy that's seen success in treating the most severe form of the disease. CRISPR Therapeutics had the treatment, brand name Casgevy, approved in the United Kingdom last month after seeing a successful global trial in which 28 out of 29 patients reported being free of severe pain. (Pedersen, 12/9)
