FDA grants Platform designation for Sarepta’s SRP-9003

US rare disease meds developer Sarepta Therapeutics (Nasdaq: SRPT) revealed that its rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the US Food and Drug Administration.

The status could help Sarepta fast-track development of future treatments using the same viral delivery system, the company noted.

“This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs,” said Louise Rodino-Klapac, chief scientific officer and head of R&D Sarepta. “The designation underscores and reinforces the consistency of the data we have seen with this AAVrh74 in multiple clinical programs and is yet another example of Sarepta’s continued commitment to accelerating the development of potentially transformative treatments for patients with rare genetic diseases like LGMD type 2E/R,” he added.