![FDA, FTC and DOJ Enforcement of Medical Device Regulations FDA, FTC and DOJ Enforcement of Medical Device Regulations](https://www.fdanews.com/ext/resources/Book-Covers-2/BFFDEMDR-COVER.png?height=200&t=1685733565&width=200)
Home » In Pair of Muscular Dystrophy Therapy Studies, Pfizer Initiates Dosing While Sarepta Hits Snag
In Pair of Muscular Dystrophy Therapy Studies, Pfizer Initiates Dosing While Sarepta Hits Snag
![pfizerlogo.gif](https://www.fdanews.com/ext/resources/test/Drug-Images/pfizerlogo.gif?t=1576044507&width=430)
January 12, 2021
Pfizer has started dosing in a phase 3 study evaluating its investigational gene therapy, PF-06939926, in young male patients with Duchenne muscular dystrophy (DMD).
The trial plans to enroll 99 boys aged 4 to 7 years across 55 sites in 15 countries. PF-06939926 received Fast-Track designation from the FDA in October.
The agency previously granted the gene therapy Orphan Drug and Rare Pediatric Disease designations in 2017.
Meanwhile, Sarepta Therapeutics reported disappointing top-line results from a phase 2 trial evaluating SRP-9001, its gene therapy for DMD.
Patients receiving the infusion did not achieve statistically significant muscle function following 48 weeks of treatment.
Upcoming Events
-
21Oct