Sarepta Therapeutics (SRPT) Eteplirsen Delayed as FDA Grasping for Data Amid Pressure - Leerink

Leerink analyst Joseph Schwartz, was the latest to weigh in on Sarepta Therapeutics (NASDAQ: SRPT) after it was notified that the FDA will not meet the PDUFA goal date of May 26 for eteplirsen.

Schwartz notes that, interestingly, the press release neither discloses nor ballparks a potential action date.

"While this announcement may supplement the volatility surrounding this stock, we maintain our cautious view on the overall NDA submission and believe the FDA will be more scientifically-driven at the end of the day," he said. "Anything but an unequivocal approval also raises the possibility of a formidable competitor re-entering the race."

He believe the FDA is grasping for any dispositive data that the drug works, given the mounting pressure from pt. advocates and politicians from Congress.

"As a scientifically-oriented organization, the FDA can't approve a drug that addresses a high unmet need pt. population purely on the safety metric," he commented. "The FDA is also a very legally-oriented organization and the law that they must follow requires substantial evidence of efficacy and not just safety; FDA should be mindful of the precedent it might set. We point out two counter arguments for eteplirsen approval - 1) eteplirsen approval sets a new threshold for NDA submission with a questionable clinical and scientific data, and 2) Dr. Janet Woodcock discussed this particular issue at our Rare Disease Roundtable (LINK) when she cautioned "that any adjustments or concessions made for one approved application will ultimately be granted to all future sponsors’ approval requests". To the latter point, recall BMRN's Kyndrisa received a CRL following their AdCom in Nov '15, which we believe to be consistent with the FDA's judicious approach to science."

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