Sarepta Therapeutics Inc. said Friday it has received a negative opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for its Exondys treatment for the rare disease Duchenne muscular dystrophy. The company had already said on its May 5 earnings call that it expected the negative opinion. Sarepta said it will now ask for a re-examination of the opinion, which would result in the assignment of a new rapporteur and co-rapporteur. “The company will also request a Scientific Advisory Group (SAG) on DMD be called so that neuromuscular specialists, experienced with working with treatments for these patients, can provide expert guidance and insight into, among other things, the validity of the external controls used and the importance of certain functional endpoints, including for instance, the relevance of meaningful slowing pulmonary decline in patients with this difficult to treat disease,” the company said in a statement. In February, Sarepta halted a trial of its golodirsen therapy due to “one serious adverse event” that it said might be related to the therapy, also used to treat DMD.Exondys was approved for use in treating DMD in the U.S. in 2016, in a decision viewed as controversial because it came despite a negative opinion from an advisory committee. It was the first treatment for the disease. DMD patients lack the protein dystrophin, which is critical to muscle function. The disorder, which is rare, mostly affects boys and typically kills patients before the age of 30. Sarepta shares were slightly higher in premarket trade Friday, but are up 206% in the last 12 months, while the S&P 500 has gained 11%.
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