Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that the FDA granted accelerated approval to Vyondys 53 (golodirsen) for the treatment of patients with Duchenne muscular dystrophy (“DMD”) who are exon 53 amenable. Vyondys 53 is Sarepta’s second approved exon-skipping RNA therapy for DMD. The company will launch the drug immediately.
The company has to conduct a post-marketing confirmatory study to convert the accelerated approval to a continued approval for Vyondys 53. We note that Sarepta is already conducting a similar study – ESSENCE. The study is currently enrolling patients and is expected to conclude by 2024.
Please note that the new drug application (“NDA”) seeking approval for Vyondys 53 had received a complete response letter (“CRL”) from the FDA in August. The FDA cited two concerns — the risk of infections related to intravenous infusion ports and renal toxicity seen in pre-clinical models — in the CRL.
Shares of Sarepta surged 29.6% in after-hours trading on Dec 12, following the approval to Vyondys 53. Investors cheered the news as the company resolved the issues mentioned in the CRL in just four months. The company’s shares have decreased 8% so far this year, against the industry’s increase of 6.3%.
The approval to Vyondys 53 is likely to boost total revenues for Sarepta. The company’s already approved DMD drug, Exondys 51, treats patients who are exon 51 amenable. Exondys 51 has generated sales of $280.7 million in the first nine months of 2019, up almost 30% year over year. The company stated that Vyondys 53 will increase eligible DMD patient population by 8% in the United States. Both the drugs combined will offer treatment options for approximately 20% of U.S. DMD patients.
The company is developing another exon-skipping RNA therapy, casimersen, for DMD patients who are exon 45 amenable. It stated in its third-quarter earnings release that it will submit a NDA for the candidate following clarity on golodirsen NDA. Thus, we expect the company to file the same with the FDA soon.
Sarepta is also developing next-generation PPMO platform candidate, SRP-5051, in DMD patients amenable to exon 51 skipping and gene therapies targeting DMD and other muscular dystrophies as well as central nervous system disorders.
Meanwhile, there are several other companies developing gene therapies for treating DMD including Solid Biosciences (SLDB - Free Report) , Audentes Therapeutics and Pfizer (PFE - Free Report) . Successful development of gene therapies for DMD will increase competition in the field.
Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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