Sarepta Dives After FDA Rejects Its Newest Muscular Dystrophy Drug
Sarepta Therapeutics (SRPT) toppled late Monday after the Food and Drug Administration rejected the biotech company’s latest Duchenne muscular dystrophy drug.
In after-hours trading on the stock market today, Sarepta stock tumbled 12% to 106. That signals a move to the lowest levels of 2019. Sarepta stock ended the regular session down 3.7% to 120.31. The FDA cited the risk of infections related to the intravenous infusion port used to give the drug, dubbed golodirsen. The agency also noted concerns of kidney toxicity in preclinical studies and in other drugs belonging to the same class, Sarepta said in a news release.
Preclinical tests — which don’t involve patients — showed golodirsen can harm the kidney when given at a 10-fold higher dose than what Sarepta used in clinical studies. There were no instances of kidney toxicity in the study that Sarepta used in its application requesting golodirsen approval.
“Over the entire course of its review, the agency did not raise any issues suggesting the non-approvability of golodirsen, including the issues that formed the basis of the complete response letter,” Sarepta Chief Executive Doug Ingram said in a written statement.
The biotech company said it immediately requested a meeting with the FDA to determine its next steps.
If approved, golodirsen would treat 8% of the population with Duchenne muscular dystrophy. DMD is a genetic disorder characterized by progressive muscle weakness. Sarepta already sells Exondys 51, which treats about 13% of patients with the disease