Doctors ready to roll with Sarepta's $300K DMD med Exondys 51. Are payers?

photo by Martin Fisch via Flickr

You know that old saw about every med school graduate winning the “Doctor” title? Something similar could apply to Sarepta’s newly approved Duchenne muscular dystrophy med Exondys 51.

Despite previous setbacks at the FDA and a highly contentious approval process--not to mention plenty of questions from the agency’s own scientists--the new DMD treatment looks poised to sell.

Top DMD specialists, along with a swath of neurologists, have told analysts at RBC Capital Markets that they’re ready to prescribe Exondys 51. Three “key opinion leaders” in DMD, in fact, plan to use the drug in “essentially all” their patients with the disease, the firm said in a Wednesday research note.

RBC talked to those three influential specialists and surveyed another 100 neurologists about their plans for the med. Only 5% of the surveyed neurologists said they don’t plan to use the drug, and 40% said they’ll use it in all or most of their eligible patients.

Another 54% said they’d try Exondys 51 in a small number of patients to see how it works before adopting it more broadly; that goes hand in hand with the fact that a significant number of the respondents--one in 5--aren’t convinced of its clinical benefit.

The DMD specialists have all been involved in clinical trials, both testing Exondys 51 and other potential therapies, and they told RBC that they’re eager to use the drug--and they pooh-poohed the surveyed doctors who were skeptical that it works. The unconvinced neurologists probably didn’t understand the available data, the specialists said. On the other hand, the FDA's own scientists raised their own questions about that available data.

The survey showed that 59% of neurologists worry that payers might raise barriers, given its $300,000 base price, which, at higher doses, would lead to much higher reimbursement costs in older patients. That’s even in a disease as rare as DMD and a patient population of just 2,000 in the U.S. In the past, insurers have typically covered rare disease drugs, many of which are priced higher than $300,000, but these days, payers are increasingly bold about setting up coverage hurdles and pushing for discounts.

The KOLs, on the other hand, don’t think payers will put up a fuss and expect that their patients will be on the drug within three months.

RBC analyst Simos Simeonidis figures payer deals will take some time, but he expects the logistics of reimbursement to be “slowly ironed out for patients in the first 2-3 quarters” after launch.

Sarepta has said it’s ready to launch the drug, with enough supply on hand for a year and work underway to scale up manufacturing further. It’s expecting eligible patients to wait 30 to 90 days for payer approval. To help patients handle their share of costs, the company is rolling out a financial assistance program, SareptAssist.

Simeonidis says he’s expecting a “very successful launch” for Exondys 51, and that, given the drug’s broad label, early safety profile and the “understandable urgency of the DMD patient community to gain immediate access to this agent, we don’t expect reimbursement to end up being a significant issue.”

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