By Bhanvi Satija
()- U.S. authorities requested Sarepta Therapeutics on Friday to temporarily stop sending out its Elevidys gene therapy product following the death of a patient with muscular dystrophy who had undergone an alternative, investigational treatment; however, the company stated it would refuse to comply with this request.
The U.S. Food and Drug Administration revealed the decision, validating a previous report, following a discussion with Sarepta during a meeting held on Friday.
Following the FDA's inquiry, Sarepta, based in Cambridge, Massachusetts, stated in a release that it will keep distributing the treatment to individuals who can move around independently, while continuing the suspension it put in place on June 15 for those who cannot walk, after notifying the FDA about an instance of severe liver damage in a non-ambulatory patient.
Sarepta stated it reached the conclusion "due to our thorough scientific analysis of the data, indicating no new or altered safety concerns among ambulatory patients."
Although the 51-year-old man suffering from limb-girdle muscular dystrophy, who passed away most recently, was not receiving Elevidys, the agency stated that both this experimental treatment and Elevidys utilize comparable genetic technologies.
The U.S. Food and Drug Administration announced it is suspending clinical studies for limb girdle muscular dystrophy because of safety issues.
In 2024, Elevidys was granted standard approval for individuals aged four years and above with a genetic mutation causing Duchenne muscular dystrophy who are still able to walk. It also obtained expedited, provisional authorization for patients suffering from this progressive muscle-degenerating condition who have lost the ability to walk, despite the treatment not achieving its primary objective in a later phase study.
Another blow came as the regulator removed the platform technology classification from Sarepta's gene therapy, a label that can expedite the approval process and is granted when a technology shows potential for various applications.
Sarepta stocks closed with a drop of 36% at $14.08. On Friday, the stock fell by more than 40%, reaching a level not seen in nearly ten years, following the announcement of the third patient's death.
The organization intensified its review of Sarepta after two young males treated with Elevidys passed away this year. Each of the three fatalities resulted from sudden liver damage and took place among patients who could not walk.
'DISAPPOINTMENT, CONCERN'
The Food and Drug Administration mentioned that it is still looking into the potential for severe liver damage leading to hospital stays and fatalities associated with gene treatments utilizing Sarepta's AAVrh74 platform.
Analysts from Wall Street believe the third fatality might cause patients to be more reluctant in using Elevidys. Patient organizations expressed concerns regarding the recent events involving Elevidys.
"Parents affected by Duchenne muscular dystrophy are dealing with a combination of sadness, worry ... and uncertainty regarding the decisions they're making for their kids or themselves," stated Debra Miller, head of the nonprofit organization CureDuchenne.
On Wednesday, Sarepta mentioned that it is collaborating with the FDA to include a cautionary notice regarding potential liver damage dangers on the labeling of Elevidys.
During an investor conference call on Friday, analysts questioned Sarepta about why it hadn’t revealed the recent patient passing on Wednesday, which coincided with the announcement of 500 job reductions and budget cuts to its limb-girdle muscular dystrophy initiative, as explained through financial considerations.
CEO Doug Ingram stated that the issue was "not significant or essential" to Wednesday's announcement, and the choice to discontinue the limb-girdle muscular dystrophy gene therapy trial was taken separately from the patient’s passing.
The firm further stated that liver problems were not an unexpected safety concern within the research. Nevertheless, certain analysts, such as those from BMO Capital Markets, expressed concerns that Sarepta's approach to sharing this information might harm the reputation of its leadership.
At minimum, two analysts questioned if additional fatalities took place within Sarepta's gene treatment initiatives. The firm stated they were unaware of any incidents outside the three that were reported.
(Reported by Bhanvi Satija from Bangalore; additional reports by Christy Santhosh; edited by Caroline Humer, Mrinalika Roy, Devika Syamnath, and Cynthia Osterman)