Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that the FDA has rejected the application for the approval of its exon 53 skipping candidate, golodirsen. The company had filed a new drug application (“NDA”) in December last year seeking accelerated approval for the candidate as a potential treatment for Duchenne muscular dystrophy (“DMD”).
The FDA issued a complete response letter (“CRL”) against the NDA for golodirsen, citing two concerns – the risk of infections related to intravenous infusion ports and renal toxicity seen in pre-clinical models.
Sarepta stated that renal toxicity in pre-clinical models was observed in doses ten times the dose used in clinical studies. The renal toxicity issue raised in the CRL were not observed in the clinical study – Study 4053-101 – which formed the basis for golodirsen NDA.
Shares of the company fell more than 13% in after-hours market on Aug 19, following the announcement. However, the stock has gained 10.3% so far this year against the industry’s decrease of 2% as demand for its sole marketed DMD drug, Exondys 51, remains strong.
The CRL comes as a surprise for investors and the company as no issues related to golodirsen NDA were raised by the FDA during the entire review period that could have led to non-approval of the candidate. In February, the FDA had granted priority review to the NDA. Moreover, on its second-quarter earnings release, the company announced that the FDA has conditionally approved the trade name of Vyondys 53 for golodirsen. Sarepta was ready to launch the drug immediately following the potential approval.
The company has one marketed drug – Exondys51 – for treating exon 51 skipping DMD. The drug has the potential to address up to 13% of the total DMD population. The FDA’s decision to reject golodirsen NDA is likely to hit the company’s near-term growth as the approval would have increased the eligible DMD patient population by 8%.
Sarepta will work with the FDA to address the issues raised in the CRL and find a path to get the candidate approved as early as possible. The company is planning to request a meeting with the FDA soon. Meanwhile, the company is evaluating the safety profile of golodirsen in an ongoing phase III study – ESSENCE – along with another DMD candidate, casimersen. The company is also evaluating golodirsen and casimersen in a phase III open-label extension study, which is currently enrolling patients.
Casimersen is an exon 45 skipping DMD candidate and the company is likely to file an NDA seeking approval for the candidate by the year end.
Apart from golodirsen and casimersen, 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 (BOLD - Free Report) and Pfizer (PFE - Free Report) .
Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Legalizing THIS Could Be Even Bigger than Marijuana
Americans spend an estimated $150 billion in this industry every year… more than twice as much as they spend on marijuana.
Now that 8 states have fully-legalized it (with several more states following close behind), Zacks has identified 5 stocks that could soar in response to the powerful demand. One industry insider described the future as “mind-blowing” – and early investors can still get in ahead of the surge.
See these 5 “sin stocks” now >>