Drug Development, Regulatory

Sarepta plays nice with the FDA, sets out plans for second accelerated Duchenne pitch by year’s end

Attempting to mend a rather tense relationship with regulators, Sarepta has wrapped up talks with the FDA on how it should move forward with its new Duchenne muscular dystrophy drug: golodirsen. With notes from the FDA in hand, Sarepta $SRPT says it plans to seek rapid approval, with an NDA filed by the end of this year.

Ellis Unger

The Cambridge, MA-based company stirred some controversy at the agency back in 2016, when the FDA approved Sarepta’s first DMD drug Exondys 51. Some regulators were skeptical of the drug’s benefit to patients, including Ellis Unger, director of the Office of Drug Evaluations. At the time, Unger warned that Exondys 51 was “essentially a scientifically elegant placebo.” He was overruled, however, and the drug came to market later that year. It’s now a decent revenue driver for the company, bringing in $57 million in Q4 sales last year.

Now, Sarepta wants a new approval for a subset of DMD patients. The experimental drug golodirsen is designed to treat patients whose condition is caused by an error in the DNA sequence called exon 53. Data on the drug shows it increases dystrophin, the protein missing in Duchenne, to 1.2% of what normal patients would produce. That’s a big improvement from the observed benefit for patients on Exondys 51, which makes 0.28% of the normal amount of dystrophin.

Doug Ingram

Sarepta wants accelerated approval of the drug, and the FDA appears to be open to it — as long as the drugmaker later produces data that correlates the biomarker with real clinical outcomes in patients. The company believes its existing Phase III trial, Essence, will do just fine to satisfy the FDA’s requirement, as it’s designed to measure dystophin production and improvements in clinical outcomes by comparing golodirsen to a placebo.

“Obviously, whether golodirsen will obtain accelerated approval is a review decision that will come after the submission and review of our NDA,” said Sarepta’s CEO Doug Ingram in a statement. “But we greatly appreciate the willingness of the Neurology Division to engage and provide clear direction to us on the steps necessary to support an NDA submission for accelerated approval.”

The best place to read Endpoints News? In your inbox.

Comprehensive daily news report for those who discover, develop, and market drugs. Join 44,900+ biopharma pros who read Endpoints News by email every day.

Free Subscription

Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

Visit Endpoints Careers ->