The U.S. FDA has filed Sarepta Therapeutics’ (SRPT) eteplirsen New Drug Application, starting the clock on a 6-month review of the drug.
The FDA will make an approval decision regarding eteplirsen on or before February 26 of next year, said Sarepta after hours on Tuesday, two months after a competing Duchenne Muscular Dystrophy drug candidate from Biomarin Pharmaceuticals (BMRN), drisapersen. That drug, developed by Prosensa Therapeutics and once partnered with GlaxoSmithKline (GSK), faces its own PDUFA on December 27th of this year, and the investing world tentatively expects an FDA Advisory Panel meeting in November to discuss the merits of BOTH drugs.
News that Sarepta had begun a rolling NDA submission this May took the stock higher by 50%, and while the application’s filing is generally a non-event in the world of biotech, eteplirsen’s development and regulatory communications have been so wrought over the course of the last three years that SRPT investors and analysts are relieved the package is finally in the hands of the FDA.
With an approval decision now a half-year away, here’s where how the Street reacted Wednesday morning:
- RBC increased its SRPT price target to $62 from $35, with an 80% probability of success for eteplirsen in the U.S. RBC sees peak sales of $720 million in 2026.
- Baird took their price target to $47 with a 70% probability of success for eteplirsen in the U.S., and 20-30% for new exon-skipping drugs.
- Roth increased their price target to $50, from $45.
- Piper Jaffray took their price target to $48, from $28, and upgraded the name to Overweight.
Also in the fourth quarter, PTC Therapeutics (PTCT) will have results from the ongoing ACT DMD phase 3 trial of Translarna (ataluren) in nonsense mutation Duchenne Muscular Dystrophy. The options market suggests a $30 move for PTCT on the binary event. Clearly, the end of 2015 and early 2016 are shaping up to be critical periods for DMD drug developers, advocates, and patients.
