WASHINGTON — For much of last fall and winter and into this spring, scientists at the Food and Drug Administration wavered over what to do about a potential new drug to slow the progression of Duchenne muscular dystrophy, a devastating disease that overwhelmingly affects boys, leaving most in wheelchairs by their teens and dead in their 20s.
In a clinical trial involving 12 boys, the drug appeared to have halted physical decline. That gave hope to parents desperate for a treatment, but regulators expressed skepticism about the reliability of the study’s results.
Convinced that this drug could succeed where others had failed, the families of patients mounted an aggressive campaign to pressure the FDA. They argued that it was up to the agency to decide whether their sons would be the last generation of boys to die from Duchenne, or the first to survive. They hired a public relations firm and bombarded Facebook and Twitter, posted online YouTube videos of boys benefiting from the drug, rounded up more than 100,000 signatures for an official White House petition, visited lawmakers on Capitol Hill, and made their case to top FDA officials in a series of face-to-face meetings.
On Monday, Cambridge-based Sarepta Therapeutics Inc. disclosed in a press release that while the FDA reiterated its earlier skepticism regarding data surrounding the drug, eteplirsen, the agency has detailed a potential path forward for the drug and indicated a willingness to consider it for accelerated approval. That news came five months after the FDA had told Sarepta that it would be ‘‘premature’’ for the company to seek approval and suggested that Sarepta might first need to conduct a larger trial, which could mean years of delay.
The company, whose stock soared by 40 percent Monday, said it plans to file a new drug application with the agency and will push forward with a larger ‘‘confirmatory’’ study that will allow more boys access to eteplirsen and give regulators more robust data about its safety and effectiveness.
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