The FDA Suddenly Approves This Drug From Sarepta Therapeutics

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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.