Just when they’d thought they’d won their biggest battle youngsters with Duchenne muscular dystrophy now have another one to fight.
One of the nation’s largest health insurance companies, Anthem, says it won’t cover the new drug Exondys51, which was approved by the Food and Drug Administration last month. The muscular dystrophy drug is controversial and the FDA approval came even though an FDA advisory committee had recommended against it.
Anthem says muscular dystrophy drug “investigational”
Anthem says it’s denying coverage because the drug is “considered investigational and not medically necessary.” A statement on the insurer’s web site says it’s uncertain whether Exondys51 has a “clinically meaningful benefit.”
To support this it points to the FDA’s labeled indications, which include a statement that a clinical benefit has not been established and continued approval is contingent continued clinical trials. Though research data has shown that that Exondys51 has increased production of the dystrophin gene in Duchenne MD patients, which should result in improved motor function in those patients, that improvement has not yet been shown in a clinical trial.
So, drug maker Sarepta Therapeutics will be required to conduct a clinical trial to confirm that the drug does actually improve those functions. If that trial fails to verify that clinical benefit, says the FDA, it may start proceedings to withdraw its approval of the drug.
Exondys51 very expensive
Exondys51 is the only drug approved in the U.S. to treat Duchenne muscular dystrophy and it’s expensive. Sarepta Therapeutics has priced it at around $300,000 a year.
Whether other insurance companies will cover the drug appears to be a mixed bag. Some, including Cigna and UnitedHealth Group, have said they will cover it. Blue Cross Blue Shield is reported to be doing the same. A spokesman for Aetna says they plan to review things further before they make a coverage decision.