Anthem Refuses To Cover Exondys 51 Citing Lack of Efficacy Evidence

The largest US health insurer, Anthem Inc, recently announced it would not cover the first FDA-approved Duchenne muscular dystrophy drug (DMD), Exondys 51 (eteplirsen; Sarepta Therapeutics) due to concerns over clinical trial data.

According to a recently released statement from Anthem, “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.”

Despite a large majority of FDA panelists questioning the efficacy of eteplirsen, pressure from patient advocates pushed the FDA to approve the drug last month. The FDA approval was based on data believed to predict a clinical benefit.

Serepta, the drug’s manufacturer, currently estimates an annual regimen of eteplirsen to cost $300,000. The company has stated that the total cost could also increase depending upon the weight of the patient. Anthem’s denial of eteplirsen is not only a large setback for Serepta but also for the FDA’s recent approval. A key pre-approval concern was whether or not eteplirsen could sufficiently produce higher levels of the protein, dystrophin which allows for increased voluntary movement, a marker of Duchenne muscular dystrophy treatment.

FDA commissioner, Robert M Califf, MD, recently commented that the study should be retracted due to his doubts about the viability of a six-minute walking test that each trial representative underwent.

The Anthem statement noted that, “Overall, both [clinical trials] failed to meet their primary endpoints of a significant improvement in six-minute walking test scores, and methodological study limitations hinder the ability to interpret the efficacy of [eteplirsen] as a disease modifying therapy for Duchenne muscular dystrophy.  Furthermore, expert consensus suggests that [the] six-minute walking test scores [are] not the best reflection of function in individuals with Duchenne muscular dystrophy.”

However, some patients will still have access to Serpta’s eteplirsen; as UnitedHealthcare and Cigna have both agreed to cover the drug. —Julie Mazurkiewicz