(Bloomberg) — A top U.S. Food and Drug Administration official has challenged reviewers to give broad approval to Sarepta Therapeutics Inc.’s gene therapy. for a rare muscle disease in children, despite a lack of data showing that it actually slows the overall progression of the disease.
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The approval was a victory for parents who had defended the controversial $3.2 million treatment, as well as for the company. Shares rose as much as 40% in New York, the most intraday in six years.
Under the expanded approval granted Thursday, Sarepta can now market the drug to the vast majority of children with Duchenne muscular dystrophy, a fatal genetic disease. It was the best possible outcome for the company, which had sought to expand approval of the treatment even after a trial had failed to clearly show it slowed disease versus a placebo. Previously, the therapy was only approved for 4- and 5-year-old children.
“Sales growth is going to be huge,” Sarepta CEO Douglas Ingram said Friday on a conference call. “Patients have waited too long for a therapy like this.”
The only provider
The new clearance means Sarepta likely has the market to itself in Duchenne muscular dystrophy gene therapy for now, as a potential competitor from Pfizer Inc. recently failed a test.
The approval came after two FDA reviewers recommended against it, according to FDA documents, noting that the results of two failed trials conducted by Sarepta created uncertainty about whether the treatment worked and in which patients. A statistical review also found that the main Sarepta trial “did not meet the criterion for success.”
But those reviewers were overruled by the FDA’s head of biologics, Peter Marks, who noted that patients taking the drug did better on other measures of disease progression. “The observations regarding secondary endpoints and exploratory endpoints are compelling,” he wrote in a memo.
According to the new decision, full approval was granted for a broad group of patients aged 4 years and older who can still walk. The agency also granted accelerated approval for use in those who cannot walk, meaning the company will need to conduct another trial to confirm benefits in this group.
The new label covers more than 80% of boys and men with Duchenne muscular dystrophy, Sarepta executives said in a conference call. The broader label will drive “significant top-line growth for the company in the near term,” Tim Lugo, an analyst at William Blair & Co., said in a note.
There are no strings attached
Brian Abrahams, an analyst at RBC Capital Markets, called the agency’s decision a “broad, almost unrelated” expansion of the label. Abrahams raised his price target on Sarepta shares from $142 to $182. Shares of the company traded up to $173.25 on Friday.
Called Elevidys, the treatment has sparked debate over the FDA’s accelerated approval process, a regulatory shortcut designed to get drugs for devastating diseases to market quickly, often based on preliminary data. When confirmatory evidence does not show clinical benefit, the FDA may withdraw such drugs from the market. While such quick approvals may help some desperate patients, critics say the system sometimes allows unproven drugs to remain on the market for years.
The FDA granted Elevidys accelerated approval in June 2023 to treat children 4 and 5 years old, the age group that appeared to have the greatest benefit in the studies.
In October, Sarepta said its confirmatory trial failed to clearly slow the disease in a year-long study of 125 young children. But secondary measures of patient movement in the trial were positive, according to the drugmaker, and the company submitted to US regulators for full approval.
Duchenne muscular dystrophy occurs primarily in about 1 in 3,500 male births worldwide. Caused by defects in a protein called dystrophin that helps keep muscle cells intact, the disease leads to severe muscle wasting and atrophy. Most patients die in their 20s, although some live longer thanks to various treatment options, such as steroids and other approved treatments that target a particular genetic mutation.
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