FDA accepts efficacy supplement to expand the Elevidys indication

The Food and Drug Administration (FDA) has accepted and filed the efficacy supplement to the Biologics License Application (BLA) for Elevidys (delandistrogene moxeparvovec-rokl) submitted by US precision genetic medicines firm Sarepta Therapeutics (Nasdaq: SRPT).

According to the Cambridge, USA-based biotech, the goals of the efficacy supplement are twofold: To expand the labeled indication for Elevidys for the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene; and to convert the Elevidys accelerated approval to a traditional approval.

The FDA has granted the Efficacy Supplement a Priority Review with a review goal date of June 21, 2024. The Agency has also confirmed they are not planning to hold an advisory committee meeting to discuss the supplement. The news saw Sarepta’s shares edge up 3.6% to $129.00 in early trading.