Sarepta Therapeutics CEO Ed Kaye described Tuesday the ups and downs the Cambridge, MA, company has had as it tries to roll out eteplirsen (Exondys 51), the first-ever approved drug for Duchenne muscular dystrophy.
At the JP Morgan Healthcare Conference in San Francisco, Kaye told attendees that more than 250 patients with the progressive, deadly disease in the U.S. have filled out “start forms” to begin the process of ultimately receiving treatment. But Kaye could not predict how many of those patients, and others to seek treatment, would get insurance coverage. Without insurance, few if any patients will be able to afford the retail price, which Sarepta has set at $300,000 a year. The drug’s price and its small amount of clinical evidence have set up a battle that Sarepta has been waging with payers since the controversial FDA approval in September.
“It’s challenging, being the first for an approved therapy,” Kaye said.
The FDA approved eteplirsen on an “accelerated” basis, which means Sarepta must provide more evidence by 2021 that it works to keep it on the market. FDA top brass were willing to give approval, overruling their staff, because eteplirsen 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 the small, flawed set of data the FDA reviewed—the results were largely from a 12-patient trial—didn’t prove that eteplirsen led to a better outcome for patients, who often die at an early age.
The drug was approved for about 13 percent of Duchenne patients with a specific genetic malfunction.
The $300,000 average price per patient, per year, is roughly in line with the high prices of several drugs for rare diseases with no other available treatments. Many observers felt insurers would accept the price, fearing a backlash if they denied a drug to a few thousand dying kids. But some insurers, like Anthem and Humana, have either denied coverage or placed restrictions on eteplirsen’s use. A recent survey by Jefferies analyst Gena Wang found several other instances of pushback from national and regional managed care organizations.
Kaye said Tuesday that some payers still consider eteplirsen an “experimental therapy” because of the conditional approval. “That had to be dismissed as a concept for them,” Kaye said, adding that Sarepta has had 50 meetings with top payers in the U.S., often explaining the disease from scratch, trying to convince payers that eteplirsen is needed to maintain patients’ function so they don’t progress. “This is not a lifestyle drug,” he said.
Sarepta reported $5.4 million revenue last quarter, short of analysts’ expectations.
Kaye expects that insurers, many of whom are reviewing patients on a case-by-case basis, will ultimately cover a majority of the patients who have submitted start forms so far.
“What we’re seeing is, when a payer says no, that’s a first step in the discussion,” Kaye said. “It’s not a definitive no.”
Payers haven’t specifically required monitoring for patients’ benefit of eteplirsen as of yet, but Sarepta—with the help of patient advocacy groups—plans to amass a database of outcome measures, reported by the patients themselves. “Payers want to know [if] patients having a better life experience because they’re on therapy,” Kaye said.
The database will track patients’ ability to maintain or regain abilities on eteplirsen, like being able to feed themselves, use a computer or electric wheelchair, or walk.
Kaye said the top Duchenne treatment centers in the U.S., which treat about 80 percent of people with the disease, are sending in start forms. But their chances for coverage, and Sarepta’s financial outlook, is hard to predict, Kaye conceded.
“We don’t have any chart, or any path to follow,” he said.
Here’s more on eteplirsen and other rare disease drugs.