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
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.
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