Anthem says it will not cover drug to treat Duchenne muscular dystrophy

Exondys 51 made by Sarepta Therapeutics-Photo by Rxdrugs.xyz

Anthem has announced it will not cover a drug approved by the Food and Drug Administration last month to treat Duchenne muscular dystrophy.

In September, the FDA approved the use of eteplirsen, called Exondys 51, made by Sarepta Therapeutics in Cambridge, Massachusetts, for the treatment of Duchenne muscular dystrophy.

Duchenne muscular dystrophy, which is among the nine types of the muscle disease, mostly affects boys and rarely girls.

Sarepta has priced Exondys 51, at about $300,000 per patient per year, according to published reports.

"The use of eteplirsen is considered investigational and not medically necessary for all indications, including but not limited to the treatment of Duchenne muscular dystrophy," Anthem said in its October 6 medical policy.

[Also: Exondys 51, new drug for muscular dystrophy, gets FDA approval]

The FDA approved the drug amid controversy. In April, the FDA's Peripheral and Central Nervous System Drugs Advisory Committee voted against approval.

The FDA approved the drug based on an increase in dystrophin in the skeletal muscle of some boys treated with the drug. The drug allows for the creation of dystrophin, the muscle protein missing in those with Duchenne muscular dystrophy.

However, Anthem said, there is still uncertainty over whether the small increase in dystrophin has a clinically meaningful benefit, including improved motor function. 

Even FDA labeling includes a statement that a clinical benefit has not been established, and continued approval is contingent upon verification in ongoing clinical trials, Anthem said.

UnitedHealth Group reportedly does plan to cover the drug.

Duchenne muscular dystrophy affects 9,000-12,000 boys in the United States. Diagnosis usually occurs around age 6 and by the age of 10, most boys have lost the ability to walk.  Few survive past their 40s.

The current standard of care focuses on the management of symptoms, including treatment with steroids to reduce the loss of muscle function.

Twitter: @SusanJMorse