Sarepta plunges on eteplirsen news
Sarepta Therapeutics Inc. (NASDAQ:SRPT) fell $8.75 (19%) to $37.68 on Wednesday after the company said FDA "would not commit to declaring dystrophin an acceptable surrogate endpoint" for accelerated approval of eteplirsen to treat Duchenne muscular dystrophy (DMD). The company plans to submit an NDA to FDA for the compound in 1H14 based on Phase IIb data. On a conference call to discuss the news, Chris Garabedian, Sarepta's president and CEO, said it is up to FDA's discretion whether to evaluate the NDA under the accelerated approval pathway, though he noted that the agency has granted full approval to drugs with limited data sets or uncontrolled studies. Sarepta said the agency did request information on the methodology and verification of dystrophin levels. Eteplirsen is a phosphorodiamidate morpholino oligomer (PMO) that induces exon 51 skipping on the dystrophin gene. The company plans to start a Phase III trial for the product in 1Q14. ...