(NBC News/Maggie Fox) — The Food and Drug Administration approved a controversial muscular dystrophy drug Monday, ignoring the advice of its advisers and delighting families and advocates who had campaigned hard for its approval.
The drug is called eteplirsen. It's aimed at a mutation that causes some cases of Duchenne muscular dystrophy, the most common type of muscular dystrophy — a degenerative disease that causes muscles to break down because cells produce faulty versions of a protein called dystrophin, or none at all.
The disease affects more boys than girls and symptoms usually show up when kids are between 3 and 5. Right now there's no treatment.
Eteplirsen, also known as exondys 51, targets a specific mutation of the dystrophin gene.
FDA's panel of expert advisers recommended against the drug's approval after an emotional, packed meeting in which children with muscular dystrophy pleaded for the drug's approval and doctors debated whether the drug helped everyone.
"I can hardly breathe," Jenn McNary, mother of two boys with muscular dystrophy in Saxtons River, Vermont, said by email.
"This is what success feels like. I can't wait to hug the boys."
The agency gave itself extra time to decide. But expectations about approval soared last week when one key FDA critic of the drug, Ronald Farkas, left the agency last week.
Farkas has been the target of social media attacks by advocates for the drug who have accused him of holding up approval
It's an unusual decision and the FDA says it's not clear whether the drug has actually helped any of the patients who have tried it.
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