FDA OKs Cambridge co.’s MD drug
Federal regulators yesterday granted tentative approval to the first drug for muscular dystrophy, following an intense public campaign from patients and doctors who pushed for the largely unproven medication created by a Cambridge biotech company.
The approval comes nearly five months after the Food and Drug Administration and a panel of outside advisers panned the drug, saying there was little evidence that it helped. But regulators faced a public backlash from patients’ families, politicians and physicians.
The FDA cleared Cambridge-based Sarepta Therapeutics’ Exondys 51 for a rare form of Duchenne muscular dystrophy, a deadly inherited disease that affects boys. It’s the first FDA approval for the degenerative condition, which causes muscle weakness, loss of movement and eventually death.
The approval was based on a company study of just 12 boys. The agency is requiring Sarepta to conduct a larger study examining whether Exondys 51 results in improved movement and function for patients. If the study fails to shows it helps, the FDA said it could withdraw the drug.
Duchenne’s muscular dystrophy is a rare disease, affecting about 1 of every 3,600 boys worldwide and usually causing death by age 25, according to the National Institutes of Health.
Pat Furlong, a patient advocate who lost two sons to the disease, called the announcement “an extraordinary win.”
“I think this is a collaborative effort that shows the FDA, companies and the patient community can work together toward a single goal, and that is improving the lives of patients,” said Furlong, founder and president of Parent Project Muscular Dystrophy, a nonprofit that helped fund travel and other expenses for those affected by the disease.