The next few months could bring the first gene therapy for hemophilia, a new type of multiple myeloma drug and an additional treatment option for a devastating muscular condition.
The Food and Drug Administration is set to make decisions on approval of all three, as well as on a closely watched arthritis pill and the first oral drug for spinal muscular atrophy, between July and September.
A regulatory OK for Roctavian, BioMarin Pharmaceutical’s experimental hemophilia A treatment, would be a particularly significant milestone — the first gene therapy for one of the world’s most well-known inherited diseases.
