The FDA Suddenly Approves This Drug From Sarepta Therapeutics

Shares of Sarepta Therapeutics were on the rise after the Food and Drug Administration (FDA) suddenly approved the company’s second Duchenne muscular dystrophy (DMD) drug.

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact. DMD is one of four conditions known as dystrophinopathies. It mainly affects children.

Shares of the stock jumped 35% in morning trading on Friday after the company received approval for Vyondys 53. About 8% of the children who have DMD have the gene mutation that Vyondys 53 treats.

Sarepta also markets Exondys 51, which was the first FDA-approved DMD treatment in 2016.

“In the span of four months, we commenced and completed the formal dispute resolution process culminating in the grant of our appeal, resubmitted our [new drug application] and obtained an approval–a great benefit to DMD patients awaiting treatment,” Sarepta president and CEO Doug Ingram stated.

A Sarepta spokeswoman said Exondys51 and Vyondys53 both have an annual list price of approximately $300,000.

“While the approval of [Vyondys 53] provides some alleviation to the P&L’s operational burn, we remain cautious of the next steps for development both for the PMO and gene therapy franchise,” remarked Oppenheimer analyst Hartaj Singh.
Sarepta shares are up 25% year-to-date.

Disclaimer: We have no position in Sarepta Therapeutics Inc. (NASDAQ: SRPT) and have not been compensated for this article.