REGULATORY UPDATE BUSINESS & FINANCE MANUFACTURING EMA, FDA Get Together On Drugs Lundbeck Takes A Singular View FDA Making A Generic Case For Eligible For PRIME And Breakthrough Toward Tackling Challenging CNS Continuous Manufacturing, p. 23 Designation, p. 19 Disorders, p. 5 Pharma intelligence Pinkwww.ThePinkSheet.com SheetVol. 78 / No. 18 May 2, 2016 informa tinue to work with FDA as it completes the Sarepta, FDA And The Dangers eteplirsen review and that it remains com- mitted to gaining approval for a Duchenne treatment. Of Strong Early Results Debra Miller, CEO and founder of the advocacy group CureDuchenne, said in an DERRICK GINGERY
[email protected] interview with “The Pink Sheet” that she wants to make sure Duchenne patients and arepta Therapeutics Inc.’s proposed advocates remain focused on dealing with Duchenne muscular dystrophy treat- the agency’s concerns. Sment eteplirsen may have been “We just work as hard as we can to help doomed by the optimism created from its Sarepta and make sure this doesn’t happen early study results, which it turned out could again and design trials that FDA will like,” not be replicated. she said. And now the company finds itself in a pre- The committee’s decision was made diffi- carious situation as it looks for a way to gen- cult by the emotional and admittedly com- erate additional scientific data that match pelling testimony from dozens of patients, the overwhelmingly positive anecdotal evi- parents and advocates (“Patients Can’t Res- dence of efficacy. cue Sarepta’s Eteplirsen” — “The Pink Sheet” FDA made no secret of the fact that it had DAILY, April 25, 2016).