Sarepta Submits BLA For SRP-9001 To Treat Ambulant Patients With Duchenne Muscular Dystrophy
(RTTNews) - Genetic medicine maker Sarepta Therapeutics, Inc. (SRPT) announced Thursday that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec) to treat ambulant patients with Duchenne muscular dystrophy.
SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Roche.
The BLA is submitted for accelerated approval based on the expression of SRP-9001 dystrophin protein, an internally shortened and functional version of dystrophin, as a surrogate endpoint reasonably likely to predict clinical benefit.
Among other things, the BLA is based on positive pre-clinical, biomarker and clinical functional results. In clinical trials, SRP-9001 demonstrated positive results at multiple time points, including one-, two- and four-years after treatment, in addition to a consistent safety profile.
For More Such Health News, visit rttnews.com
Quest Diagnostics Says FDA Grants Breakthrough Designation For Adeno-associated Virus Test
FDA OKs Elevidys As First Gene Therapy To Treat Duchenne Muscular Dystrophy
Sarepta Therapeutics Updates Of BLA For Sarepta's Duchenne Muscular Dystrophy Drug Candidate
Sarepta Therapeutics Says FDA Lifts Clinical Hold On SRP-5051 To Treat Duchenne Muscular Dystrophy
U.S. Consumer Prices Increase In Line With Estimates In August
IEA Cuts Oil Demand Forecast; Canadian Markets Eye Recovery
Canadian Dollar Falls As Oil Prices Edge Lower
