Dealing a blow to Sarepta Therapeutics’ launch efforts, leading U.S. insurer Anthem has decided not to cover the pricey Duchenne muscular dystrophy drug Exondys 51. Anthem cited "uncertainty" about whether the drug actually works, in the wake of an FDA approval hotly contested within the agency and criticized by some in the scientific community.
In a medical policy notice posted to its website, Anthem said that “clinical benefit of treatment for DMD with eteplirsen, including improved motor function, has not been demonstrated.”
That's exactly what many of the FDA's internal reviewers and expert panelists said when evaluating Sarepta's data on the drug. The agency approved Exondys 51 last month, despite those efficacy questions.
Anthem’s decision to block Exondys 51 is a break with its fellow insurance giants, at least so far. UnitedHealthcare says it plans to cover the drug, a spokesperson told FiercePharma. Aetna plans to conduct a “full clinical review” to determine whether and how to cover it.
The pharmacy benefits manager Express Scripts, for its part, will have a committee review the med "in the near future," according to a spokesperson.
The FDA's own internal review documents detailed many issues with the drug's supporting data, and after a raucous meeting to consider the med, panelists voted 7-3 against approval, with three abstaining, and 7-6 against accelerated approval, a designation that comes with requirements for follow-up study.
But those questions about the drug's efficacy drew intense fire from patient advocates, who lobbied hard for its approval. As the FDA review process neared its close, some agency higher-ups still opposed its approval, but last month, the agency's CDER chief, Janet Woodcock, overruled them.
With that approval finally in hand, Sarepta priced Exondys 51 at around $300,000 per patient per year, an amount analysts saw as reasonable given the small group of target patients. Exondys 51 is designed for certain DMD patients with a confirmed mutation of the dystrophin gene. About 1,200 to 1,500 boys in the U.S. could benefit from the approach, Anthem said.
Before the Anthem decision, some DMD specialists said they plan to prescribe the drug in “essentially all” of their eligible patients, according to a note issued late last month by analysts at RBC Capital Markets. Only 5% of specialists said they don’t plan to use it.
At the time, the analysts cited “key opinion leaders” who didn’t believe payers would put up barriers to access, adding that their patients would likely be on the drug within three months. Anthem’s decision could put a check on that sentiment and on the efforts to get patients access to the drug.
As part of its approval, the FDA required a two-year confirmatory trial for the drug. Anthem added in its notice that “establishment of a clinical benefit is warranted in on-going clinical trials.”
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