Shocking FDA rejection casts new doubt on the future of gene therapy

In a surprising turn of events, the FDA yesterday rejected BioMarin Pharmaceutical's gene therapy for hemophilia — a decision that might stand to complicate the future of other one-time gene therapies for rare diseases. The agency said the company needed to provide two more years of data to show that the benefits of the drug won't wane over time. The FDA has approved other gene therapies with shorter study lengths, but there's now a question of whether the agency will expect longer-term data for other gene therapies going forward.“I would not yet go as far as saying this signals a higher bar for gene therapies at FDA, Ramsey Baghdadi, co-founder of the regulatory consulting firm Prevision Policy tells STAT. Read more here.