Alnylam vows value-based pricing, financial help with $450K Onpattro launch

“Game-changing” and “a tremendous achievement” are just a couple of the ways analysts and regulators described Friday’s approval of Alnylam RNAi drug Onpattro. But the biotech still has work to do if it wants to capitalize on the drug’s launch.

First off, Alnylam has to make sure patients can get their hands on Onpattro, a rare-disease therapy that treats peripheral nerve disease caused by the abnormal protein condition hereditary ATTR amyloidosis—and bears a hefty price tag to match. The drug is rolling out with a list price of about $450,000, which Alnylam expects to yield $345,000 per year after rebates and discounts.

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Alnylam priced Onpattro, in part, to “obtain fair reward for a new class of medicines that has the potential to transform the treatment of diseases,” it said on a fact sheet, pointing to the 16 years of R&D required to bring the first RNAi therapy—which works by “silencing” mRNA inside cells to stop production of disease-causing proteins—to market.

The company is working out value-based pricing agreements with “leading commercial insurers,” it said, noting that it’s already agreed “in principle” on the structure of a pact with Harvard Pilgrim Health Care and other payers. “Partnering with payers on these agreements is intended to … help accelerate coverage decisions for patients,” Alnylam noted.

It’s also offering copay assistance for eligible patients and doling out the drug for free to uninsured patients or those whose insurance won’t cover it. And then there’s Alnylam Assist, a patient-support program that includes insurance and financial assistance case managers.

According to Alnylam, between 10,000 and 15,000 U.S. patients could be candidates for Onpattro, meaning there’s blockbuster potential in the drug’s future. But first, it’ll have to get the word out. Fewer than 3,000 of those patients are diagnosed today, Evercore ISI analyst Steven Breazzano wrote in a Friday note to clients.

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To do so, Alnylam launched a disease-awareness campaign earlier this year, and the company is staffing up, big time. It plans to double its employee head count by 2020, the Boston Business Journal recently reported.

But for now, at least, reps won’t be able to tout cardiac safety data from Alnylam’s phase 3 Apollo study. “Near the end of the day the review process labeling discussions intensified and at the end of the day FDA did not believe that ALNY had firmly established the cardiac benefit,” Breazzano wrote, adding that the drugmaker “continues to have a productive dialogue with the FDA and will continue to understand what else might be required.”

To Breazzano and his colleagues, the omission “doesn’t strike us as a deal breaker by any means,” but it could mark a point of differentiation for a future competitor—namely, Pfizer’s tafamidis. That drug “is likely to enter the market in 2019 with a broad label," he wrote, covering both ATTR and its sister disease, wild-type TTR, and could also include polyneuropathy.