A few weeks ago, Xconomy spoke with analysts and patient advocates who considered it unlikely that insurers would balk at a potential price of $300,000 a year for eteplirsen (Exondys 51), the first approved drug for Duchenne muscular dystrophy. That’s largely been the case so far, but today a significant outlier emerged—Anthem, one of the largest health insurers in the country.
On Friday, Indianapolis-based Anthem (NYSE: ANTM) disclosed that it won’t cover eteplirsen, calling the medication “investigational and not medically necessary” for the treatment of Duchenne, a progressive and fatal genetic disease. According to a spokesperson for the drug’s owner Sarepta Therapeutics, other large insurers UnitedHealthcare and Cigna have already said that they will cover the drug, which was approved by the FDA on Sept. 19. Anthem is the first big insurer to say no. The news sent shares of Cambridge, MA-based Sarepta (NASDAQ: SRPT) down 6 percent.
In its decision, Anthem cited many concerns that made eteplirsen’s approval last month controversial. The agency approved the drug on an “accelerated” basis because it appears to boost the levels of the muscle-protecting protein dystrophin, which Duchenne patients lack. The FDA said at the time that the dystrophin increase was “reasonably likely to predict a clinical benefit.”
But it’s still unclear how much of a dystrophin boost truly helps improve the health of Duchenne patients. Their muscles weaken until they can no longer walk, and they often die at an early age. And the small, flawed set of data the FDA reviewed—the results were largely from a 12-patient trial—didn’t conclusively prove that eteplirsen led to a better outcome for patients. Post-approval trials are underway to prove that benefit.
In its statement, Anthem said that while it recognizes the severity of Duchenne and “empathize[s] with the pain and suffering” of patients and their families, it’s committed to ensure that its members “have access to safe and effective medical technologies supported by evidence that they improve health outcomes.”
“In reviewing new drug treatments, technologies and devices, we rely on scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community,” Anthem said in the statement. “In reviewing the medical literature, Anthem’s medical policy and technology assessment committee, a majority of whom are external physicians, determined that [eteplirsen] failed to show it improves health outcomes, and therefore it is not a covered benefit for our members.”
Anthem added that it “eagerly await[s] results of additional studies,” including the FDA-mandated post-approval studies underway, and it plans to review the data when available. Sarepta is supposed to report data from its post-approval trials by 2021.
In response, Sarepta released a statement Friday that implied that Anthem’s decision might not be final. “We cannot comment on ongoing reimbursement discussions but we do have a robust team that is actively educating payers on the safety and efficacy of [eteplirsen],” Sarepta’s statement read. “We are grateful to the many insurance carriers that have quickly decided to reimburse [eteplirsen] and accelerate getting this medicine to patients as soon as possible.”
The FDA rejected two Duchenne drugs this year before approving eteplirsen, and many scientists within the agency argued eteplirsen should be rejected as well. They feared that a positive ruling would be tantamount to the agency caving to intense pressure from political figures and patient advocates despite the drug’s flimsy data. Janet Woodcock, the FDA’s top drug evaluator, fought for approval, and though others appealed her decision, she was backed by commissioner Robert Califf, who ultimately gave eteplirsen the green light.
Insurers now stand as the final gatekeepers between Sarepta’s drug and patients clamoring for a treatment. The day of approval, Sarepta said eteplirsen’s price would be based on a patient’s weight but could average roughly $300,000 a year, and while that’s in line with other rare disease drugs, the price currently comes without a proven benefit.
Still, outsiders polled by Xconomy a few weeks ago expected little pushback from payers, citing a likely avalanche of bad press that would follow. Only a fraction of patients are amenable to eteplirsen treatment—about 13 percent of Duchenne patients, an estimated 5,000 total in the U.S. and Europe, according to analyst reports—and they have no other effective treatment options.
“That [Anthem decision] will have quite a substantial impact,” says Jeremy Levin, the CEO of New York-based Ovid Therapeutics, which is developing drugs for rare brain disorders. Levin, who is also the former head of generics giant Teva Pharmaceutical Industries, is concerned about the ramifications of payers deciding the merits of a drug that has already been approved by authorities.
“One needs to be very careful about who determines what is medically necessary, and what is a real drug,” Levin said. “The FDA tells you whether the drug is effective or not,” and second guessing that judgment is akin to “second guessing what is or isn’t a drug.”