Pfizer Wins FDA Fast Track for Duchenne Muscular Dystrophy Treatment
Pfizer has secured a Fast-Track designation from the FDA for its investigational gene therapy PF-06939926 for the treatment of Duchenne muscular dystrophy (DMD).
DMD is a life-threatening disease brought about by mutations in the gene-encoding dystrophin protein.
The fast-track approval was based on phase 1 trial data suggesting that intravenous administration was effective in sustaining dystrophin levels over the course of a year.
Pfizer plans to launch phase 3 testing immediately, a company spokesperson said.