© Reuters. Sarepta (SRPT) Plunges 20% as Surprise FDA AdComm for SRP-9001 Adds Risk
By the Investing.com team
Actions of Sarepta Therapeutics (NASDAQ:) fell 20% in pre-open trading on Friday after the company announced a surprise FDA AdComm for SRP-9001, its investigational gene therapy for the treatment of Duchenne muscular dystrophy. . The news comes just weeks after management indicated there would be no AdComm.
While the news adds a layer of risk, most analysts still expected FDA approval by the PDUFA date of May 29.
“FDA’s decision to hold a public advisory committee meeting on SRP 9001 BLA is a departure from the position communicated at the mid-term meeting,” said Doug Ingram, Sarepta’s president and CEO. “While we are disappointed that we must communicate a change in decision after our earlier statement on the matter, we are not disappointed with the decision to hold an advisory committee.” He added that they will be “well prepared.”
Reacting to the news, Credit Suisse analysts believe the decision could be related to OTAT’s recent reorganization into the super office OTP. In addition, SRP-9001’s status as “one of the first surrogate endpoint-based gene therapy BLAs along with CBER’s leadership focus on biomarker-driven accelerated approval pathways, especially in rare diseases, may also play a role.” a paper”. Analysts don’t think security is an issue and see a high probability of approval. That being said, the “potential for stock price swings around 3 key events, i.e., Briefing Papers, AdCom and PDUFA,” sees the company lower its price target to $139 from $144 while maintaining a rating. Neutral.
Oppenheimer analysts said that while AdComm adds a layer of risk, they “continue to point to extensive scientific work behind the SRP-9001 gene cassette and rh74AAV vector, the compelling safety/efficacy profile of SRP-9001 to date (>100 children with DMD) and The leading experience in SRPT manufacturing.” Analysts continue to expect a 75% approval probability and remain bullish with an Outperform rating and a $180 price target.
“We believe that the comprehensive data releases from SRPT, the large unmet need for disease-modifying therapy in DMD, and the endorsement of KOL should support a positive outcome,” they commented. “Having said that, we believe that more than 80% of committee members voting in favor of the proposed question should result in a positive PDUFA. Conversely, less than two-thirds support could indicate a higher likelihood of a CRL “.
