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The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval despite concerns from some government scientists about the treatment’s ability to help boys with the inherited disease.
The Food and Drug Administration approval provides a new option for some patients with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes weakness, loss of mobility and early death. It almost always affects males.
Drugmaker Sarepta Therapeutics said it would charge $3.2 million for the one-time treatment, slightly less than a $3.5 million gene therapy for hemophilia launched last year. Like most medicines in the U.S., the cost will be mostly paid by insurers — not patients — including private plans and government programs.
The FDA OK’d the treatment only for children ages 4 and 5, based on study results showing the therapy helped produce a protein needed for muscle growth, which is missing in boys with the condition. The gene therapy had been studied in children up to age 7.
Patients, physicians and parents pushed for the therapy’s approval at a public meeting in April, sharing videos of boys running, riding bikes and doing sports and other activities, which they attributed to the treatment.
But FDA scientists detailed a long list of concerns with the company’s research, particularly a mid-stage study that the company submitted for FDA review. Overall, it failed to show that boys who received the therapy performed significantly better on measures like standing, walking and climbing than those who got a dummy treatment, although the results were better in younger kids.
Still, the FDA’s outside experts voted narrowly in favor of making the gene therapy available on a preliminary basis, noting the deadly nature of Duchenne and the risk of delaying a potentially beneficial treatment. The vote was non-binding, but the FDA often uses such recommendations to bolster its decisions.
The gene therapy was the latest treatment OK’d through the FDA’s fast-track route, which allows drugs to launch based on early results, before they’re confirmed to benefit patients.
This article was provided by The Associated Press.