Sarepta looks to get 2nd experimental Duchenne drug approved

Mar 12, 2018

Sarepta Therapeutics is working quickly to get a new drug approved to treat Duchenne muscular dystrophy, a rare, inherited disease that causes muscles to weaken.

According to the company, the FDA said it will welcome an application for the drug, even though Sarepta’s first treatment for the disease caused controversy at the agency. When that treatment, called Exondys 51, was approved in 2016, a top FDA official reportedly warned that it was nothing more than an “elegant placebo.”

Despite the agency’s skepticism with Exondys 51, the agency said it would fast track the new treatment, golodirsen, if the company could produce convincing data showing that it is capable of boosting levels of a muscle protein typically absent in patients with Duchenne.

Sarepta said it hopes to submit its application for approval to the FDA by the end of this year.

Read the full Stat News report.