The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government...
WASHINGTON — The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday 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. Sarepta’s IV treatment delivers a replacement gene for the one that is mutated in boys with the condition.
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. The FDA advisers who backed the drug also seemed reassured that data from an ongoing 120-patient late-stage study is expected to wrap up late this year. If the results don’t show a benefit, the FDA has the option to revoke the approval.
Agency leaders have also pledged to use “regulatory flexibility” when considering drugs for rare diseases, such as Duchenne, which affects about 1 in 3,300 boys in the U.S. Most people with the condition do not live past their 20s.
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