US FDA grants Fast Track Designation to gene therapy for Duchenne muscular dystrophy (DMD)
The designation was based on preliminary data from a Phase 1b study, in which PF-06939926 demonstrated durable and statistically significant improvements in multiple efficacy endpoints measured 12 months following infusion, in 9 boys aged 6-12 with DMD.
Source:
Biospace Inc.
SPS commentary:
DMD is a life-threatening X-linked disease caused by mutations in the gene encoding dystrophin, which is required for proper muscle membrane stability and function. PF-06939926 is a recombinant adeno-associated virus serotype 9 capsid carrying a shortened version of the human dystrophin gene under the control of a human muscle-specific promotor.