(VOR News) – Sarepta Therapeutics announced late Friday evening that it will not comply with the Food and Drug Administration’s (FDA) request to halt all gene therapy shipments.
This request was made after a third patient who was undergoing one of the company’s muscle dystrophy treatments passed away.
The extremely unusual action is the latest in a string of events that have been hurting the company’s stock for weeks and have lately forced the company to lay off 500 employees.
Elevidys’ FDA violations jeopardize its future availability.
In a statement released Friday night, the Food and Drug Administration (FDA) said its representatives met with Sarepta and asked the business to halt all sales.
The government has the power to remove medications from distribution, but “the company refused to do so.” The regulatory process is onerous and can take months or even years to finish. Instead, the government will typically make a request in a less formal manner, and companies will nearly always comply.
“While we are committed to ensuring that unmet medical needs are met by accessing drugs, we are not apprehensive about taking immediate action when a serious safety signal is identified,” said Food and Drug Administration Commissioner Marty Makary in a statement.
Even though Elevidys has been under criticism since its approval in 2023, it is the first gene therapy to be licensed in the United States for the treatment of Duchenne’s muscular dystrophy, a deadly disease that affects males and causes muscle atrophy.
The one-time treatment was quickly approved, even though several FDA scientists were skeptical about its effectiveness. Last year, the Food and Drug Administration (FDA) fully approved the therapy, allowing it to be used on patients 4 years of age and older, including those who were unable to walk.
Prior to its availability, only younger patients who were still mobile could use it. Sarepta said Friday that its scientific review revealed no new or changed safety signals for younger, early-stage Duchenne’s disease patients.
The organization has stated that it will continue to make the drug available to these individuals. In a statement, the group said, “We anticipate further discussions and the exchange of information with the FDA.”
Sarepta delayed the drug’s administration to older boys suffering from Duchenne’s disease, a disorder that gradually weakens the muscles and bones and causes early death. The medicine was supposed to go to these boys. In reaction to the deaths of two male teenagers undergoing therapy, the decision was made.
Additionally, the group announced Friday the death of a third patient, a 51-year-old who was receiving experimental gene therapy and was enrolled in a clinical study for a specific type of muscular dystrophy.
Sarepta notified the FDA of the death. Experiment has been suspended.
The Food and Drug Administration said Friday. In the manufacturing process, Sarepta claims that the gene therapy implicated in the event uses “a different dose” and a “different process” than Elevidys.
Liver damage was listed as an adverse effect in the Sarepta prescription materials, and it was linked to the deaths of all three individuals. At the beginning of this week, Sarepta declared that it would be cutting a third of its employees and adding a serious warning to the drug.
Due to the business’s omission to mention the third patient death in the news release or conference call used to make the revisions, Wall Street analysts were very critical of the company.
As of Friday, the company’s stock had dropped more than 35%, ending at $14.07.
Location: Cambridge, Massachusetts Since 2016, the FDA has approved three more drugs for Duchenne’s disease that Sarepta has developed, although none of them have been proven to be effective.
Concern has long been raised about the company’s inability to finish several studies that were necessary to get the FDA to fully approve its drugs.
SOURCE: USN
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