Sarepta Therapeutics Inc. (NASDAQ: SRPT) shares jumped on Friday after the firm announced that the U.S. Food and Drug Administration (FDA) has approved Vyondys 53 (golodirsen) for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping.
A Complete Response Letter (CRL) was previously issued in August for Vyondys 53. However, Sarepta made a formal dispute resolution and the FDA’s Office of New Drugs granted the company’s appeal. Sarepta then resubmitted its NDA to the Review Division, which worked to review and approve Vyondys 53.
According to estimates, Vyondys 53 may treat up to 8% of the DMD community, representing those patients who have a confirmed exon 53 amenable mutation.
This indication is based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with Vyondys 53, which is reasonably likely to predict clinical benefit for those patients who are exon 53 amenable.
Consistent with the accelerated approval pathway, the continued approval of Vyondys 53 may be contingent on confirmation of a clinical benefit in this post-marketing confirmatory trial.
Doug Ingram, president and CEO of Sarepta, commented:
In the span of four months, we commenced and completed the formal dispute resolution process culminating in the grant of our appeal, resubmitted our NDA and obtained an approval – a great benefit to DMD patients awaiting treatment. This unprecedented timing could not have been achieved without the commitment of the Review Division under the leadership of Dr. Billy Dunn, and the Office of New Drugs, which expeditiously heard and granted our appeal. Along with the DMD community, we owe our gratitude to both the Review Division and the OND for their objective, evidence-based approach to this review, for their fairness, and for the sense of urgency with which they addressed and resolved the CRL and granted this approval.
Shares of Sarepta were last seen up about 32% at $132.78 on Friday, in a 52-week range of $72.05 to $158.80. The consensus price target is $183.09.