FDA turns down Sarepta’s DMD drug over safety concerns

pharmafile | August 20, 2019 | News story | Manufacturing and Production |  DMD, FDA, approval safety, pharma, sarepta 

The FDA has declined to approve Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) drug, citing safety concerns including risk of infection and kidney infection.

Sarepta’s treatment golodirsen was expected to generate almost $400 million in sales over five years. Shares in the Cambridge, Massachusetts-based firm dropped 12.7% on announcement of the news.

Golodirsen, Sarepta’s second drug, had shown kidney toxicity in animal trials. However Sarepta noted the doses used in animla trials were ten-fold the amount than those tested in humans

“We are very surprised to have received the complete response letter this afternoon. 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,” said Doug Ingram, president and chief executive officer, Sarepta.

“We will work with the Division to address the issues raised in the letter and, to the fullest extent possible, find an expeditious pathway forward for the approval of golodirsen. We know that the patient community is waiting.”     

Sarepta’s first drug Exondys 51, also for DMD, costs on average $300,000 a year. The firm had expected to charge patients a similar amount for golodirsen.

Louis Goss