STAT Plus: In a shocking decision, FDA rejects BioMarin’s gene therapy for hemophilia
The Food and Drug Administration on Wednesday rejected a one-time therapy for hemophilia A, delaying what would have been a major medical milestone in a decision that upended the expectations of doctors, patients, and Wall Street.
The FDA’s move means the gene therapy, which promised to revolutionize the treatment of the bleeding disorder, will be delayed until at least 2022, according to BioMarin, the treatment’s manufacturer.
