On Thursday, the US gave preliminary clearance to the first gene therapy for a fatal form of muscular dystrophy despite reservations from some government scientists about the treatment’s potential to benefit boys with the genetic condition.
For some Duchenne muscular dystrophy patients, the Food and Drug Administration’s approval opens up a new treatment option.
Treatment For Young Boys With Muscular Dystrophy
Duchenne muscular dystrophy is a rare, muscle-wasting condition that results in weakness, loss of mobility, and early death. Males are almost usually affected.
A $3.5 million gene therapy for hemophilia that was introduced last year would cost somewhat less than the one-time treatment, according to drugmaker Sarepta Therapeutics, which predicted it would cost $3.2 million.
Like the majority of US medications, insurers, including private plans and government programs, will cover the cost primarily rather than consumers.
Based on research data demonstrating the therapy helped create a protein needed for muscular growth that is lacking in condition-affected boys, the FDA approved the therapy only for children ages 4 and 5. Up until age 7, children were examined for gene therapy.
At a public conference in April, supporters of the therapy included patients, doctors, and parents.
They showed films of boys engaging in sports, biking, running, and other activities that they claimed were made possible by the therapy.
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FDA Scientists Identify Issues Over Study Data
However, a large list of issues with the company’s research were highlighted by FDA scientists, particularly a mid-stage study that the company submitted for FDA approval.
Overall, the study did not demonstrate that boys receiving therapy outperformed boys receiving a dummy treatment in activities including standing, walking, and climbing, while the outcomes were better in younger children.
Despite this, the FDA’s outside experts narrowly decided in favor of releasing the gene therapy on a trial basis, citing the lethal nature of Duchenne and the danger of postponing a possibly curative treatment.
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