BRIEF—Setback for Sarepta's DMD drug in Europe

US drugmaker Sarepta Therapeutics last revealed that it has received a negative trend vote following its European Medicines Agency/Committee for Medicinal Products for Human Use (CHMP) oral explanation for its key Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen) and it will request a re-examination and Scientific Advisory Group to be convened.

Doug Ingram, Sarepta’s president and chief executive, said along with the company’s first-quarter financials presentation: “Unfortunately, in addition to our successes in the first quarter, we also have had a delay in our effort to bring eteplirsen to patients in Europe who could potentially benefit from it. I could not be prouder of our Sarepta team and the team of experts who spoke on behalf of eteplirsen at the CHMP oral explanation last week. The rigorous work that was done to prepare for the hearing only strengthened our resolve that eteplirsen should urgently be made available to those waiting in Europe. Unfortunately, the CHMP’s trend vote was negative.”

He continued: “Based on discussions with CHMP representatives, it is our understanding that the CHMP did not conclude that eteplirsen is ineffective for exon 51 amenable patients, but rather that Sarepta has not yet met the regulatory threshold for conditional approval, in part due to the use of external controls as comparators in the studies.”

First quarter 2017net revenues of Exondys 51, the company’s only marketed product that was finally granted regulatory approval in the USA in September 2016, totaled $64.6 million.