Oct 7 (Reuters) - Health insurer Anthem Inc said it would not cover the first U.S. Food and Drug Administration-approved Duchenne muscular dystrophy (DMD) drug, developed by Sarepta Therapeutics Inc, calling it "investigational and not medically necessary".
Bowing to pressure from patient advocates, the FDA approved the drug, Exondys 51, last month, even though an outside panel of experts and the agency's own reviewers questioned its efficacy.
The accelerated approval is based on data believed to predict a clinical benefit. Sarepta has to prove that benefit in a subsequent clinical trial.
Anthem, the second-largest health insurer in the United States, said on its website on Friday the drug's clinical benefit, including improved motor function, had not been demonstrated. (bit.ly/2e9qGhu)
"Exondys 51 failed to show it improves health outcomes, and therefore it is not a covered benefit for our members," Anthem spokeswoman Leslie Porras said in an emailed statement on Friday.
The drug treats a subset of patients with DMD, a rare, progressive genetic disorder that hampers muscle movement, eventually killing most patients by the age of 30.
The subset includes about 13 percent of all DMD patients, or some 1,300 to 1,900 in the United States.
Sarepta has priced Exondys 51 at about $300,000 per patient per year. The Cambridge, Massachusetts-based company's stock was down about 8 percent on Friday.
"We hope that insurers will understand the unmet need in Duchenne and will allow physicians to use their professional opinions to prescribe and monitor the benefits of all approved drugs," said Debra Miller, CEO of non-profit organization CureDuchenne, which provided Sarepta early funding to develop the drug. (Reporting by Natalie Grover in Bengaluru and Caroline Humer in New York; Editing by Shounak Dasgupta)