logo
  

Sarepta Submits BLA For SRP-9001 To Treat Ambulant Patients With Duchenne Muscular Dystrophy

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

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
Japanese entrepreneur Yusaku Maezawa has selected ten crewmembers, comprising artists, content creators, and athletes, to fly with him on SpaceX's Starship around the Moon on the dearMoon mission. SpaceX's first private lunar flight with dearMoon crew, including two backups, is planned to take place in 2023. Maezawa announced the crew for the mission in a YouTube video and a tweet. SpaceX, owned by Elon Musk, has launched 40 satellites of British communications network OneWeb into orbit. From NASA's Kennedy Space Center or KSC in Florida, Falcon 9 rocket on Thursday evening carried the satellites. The Falcon 9's first stage separated, and it then returned to Earth to make a landing on a SpaceX pad at the nearby Cape Canaveral Space Force Station. Google, owned by Alphabet Inc., plans to merge its teams working on Maps products and the mapping service Waze with effect from December 9, reports said. The decision to consolidate processes comes as the search giant is facing pressure to streamline operations and cut costs. The restructuring is expected to reduce overlapping work across the Waze and Maps products.
Follow RTT