Update: This post has been updated to include comments from a spokesperson from Humana, who reached out following publication.
- Major U.S. insurer Humana has decided to insure Sarepta's newly approved drug for Duchenne muscular dystrophy, but only if prior authorization is obtained, according to a pharmacy coverage policy document posted to Humana's website.
- Humana will consider Exondys 51 (eteplirsen) medically necessary provided the DMD patient has a confirmed genetic mutation amenable to exon 51 skipping and is ambulatory, the document said. Initial approval will cover six months of treatment.
- Despite the controversy over Exondys 51's approval by the Food and Drug Administration, most large insurers have opted to cover the drug. So far, Anthem has been the only exception, judging Exondys 51 to be "investigational and not medically necessary."
The FDA's decision to conditionally approvel Exondys 51 continues to divide experts, even as many in the patient community welcome the first disease-modifying treatment approved for DMD in the U.S.
Exondys 51 was granted accelerated approval based on a surrogate endpoint — in this case improvement in production of the dystrophin protein — rather than measured clinical benefit. While FDA reviewers were generally in agreement that dystrophin production was an acceptable surrogate endpoint, senior staff were conflicted over how much of an increase was need to reliably predict clinical benefit.
Sarepta's clinical evidence showed some increase in dystrophin production among studied patients, but the effect was small and FDA reviewers questioned the company's methods for gathering data.
Following approval, Sarepta priced Exondys at approximately $300,000 per patient per year, although that figure is based on patient weight.
"Humana’s Pharmacy and Therapeutics (P and T) Committee determined that Exondys 51 meets an unmet medical need as there is a lack of effective alternative therapy for the treatment of Duchenne Muscular Dystrophy," a spokesperson for the insurer said.
"The drug requires an approval on a member-by-member basis where we look for appropriateness of the treatment per FDA label."
Humana recommended a dosing of 30 milligrams per kilogram of body weight once weekly.
After the six months covered by initial approval, the insurer can permit continuation of treatment on Exondys 51 if the patient remains ambulatory, according to the document.