In biotech news today, a lead producer of genetic medicine for rare diseases saw its stock plunge after the U.S. Food and Drug Administration (FDA) rejected a key drug candidate.
Sarepta Therapeutics (NASDAQ: SRPT) announced today that it received a Complete Response letter from the agency, completely rejecting a proposal for the accelerated approval of their drug, golodirsen, which would be injected into patients to treat Duchenne muscular dystrophy.
Specifically, the FDA’s response focused on two main areas. Firstly is the risk of possible infections through intravenous infusion ports. The second issue, arguably the biggest, comes down to the fact that there has been an increase in renal toxicity from pre-clinical models for golodirsen, rates that were ten times higher than the dose used in other clinical studies without the drug.
Although renal toxicity wasn’t seen in Study 4053-101, which the application for golodirsen was based, it’s enough of a worry for the FDA that they’ve rejected the drug for the moment.
“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.”
The biotech company went on to say that it immediately requested a meeting with the FDA to determine what they can do moving forward. Golodirsen, if approved, would end up treating 8 percent of the total patient population suffering from Duchenne muscular dystrophy, a genetic disorder where one’s muscles become increasingly weaker.
Sarepta already sells another drug called Exondys 51, which treats around 13 percent of patients with the disease, while the approval of golodirsen would be a second option for patients that aren’t eligible one way or the other to take Exondys.
Shares of the company have fallen by over 17 percent so far in the day in response to the news. At its worst point, the company dipped into the negative by 25 percent, jumping back by almost the same amount, before ending the day down 17 percent all in a matter of half an hour in after-hours trading. Over a six-month horizon, Sarepta’s stock price has been largely erratic, jumping anywhere between $110 and $160 per share.
Sarepta Therapeutics Company Profile
Sarepta Therapeutics is a biotechnology company focused on treating rare, infectious, and other diseases. It targets a broad range of diseases while focusing on rapid development of its drug candidates. Sarepta’s strategy involves proprietary RNA-targeted technology platforms to be used for developing novel pharmaceutical products to treat a broad range of diseases and address key unmet medical needs. The company uses third-party contractors to manufacture its product candidates. Most of Sarepta’s product candidates are at an early stage of development. – Warrior Trading News
