JPM: Alnylam CEO looks to action-packed 2020 on the heels of Givlaari approval

Alnylam's Onpattro posted 20% quarter-over-quarter sales of $56 million in the fourth quarter. (Alnylam)

Still basking in an FDA approval for its second RNAi therapy, Alnylam is thinking big about just how far it can go with its new and impending launches.

With two meds on the market and four late-stage candidates–-including two partner drugs––approaching approval decisions in the next 12 to 24 months, Alnylam CEO John Maraganore said Monday he predicts an action-packed year in 2020. 

If the drugmaker does make the jump from two marketed meds to six in the coming years, Alnylam could be on its way to a "top five" position in biopharma based on the strength of its launches and budding portfolio, Maraganore said at the annual J.P. Morgan Healthcare Conference in San Francisco.

But first, the company has to rev up its brand-new Givlaari and slow-to-start debut med Onpattro—and according to Maraganore, that's on track.

"We’re very pleased with our commercial execution for 2019 and expect this to continue for 2020 and beyond," the CEO said.

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Alongside Givlaari, an RNAi therapy for rare liver disease acute hepatic porphyria (AHP) approved in November, Alnylam also markets its first approved RNAi therapy Onpattro for peripheral nerve disease caused by hereditary transthyretin-mediated amyloidosis.

Givlaari launched at an average annual cost of $575,000 per patient based on a list price of $39,000 per vial, the drugmaker said. However, Alnylam rolled out a novel reimbursement strategy for payers, utilizing "prevalence-based adjustments" that offer rebates if the prevalence of AHP patients on a given plan is higher than current estimates.

In his comments to investors, Maraganore touted that reimbursement plan as a strong commercial driver for Givlaari.

"The goal of all is to really ensure that all incentives are aligned and that we can ensure strong patient access for this medicine," Maraganore said.

Onpattro, which the FDA approved in August 2018, had a slow start out of the gate but has picked up some steam recently, hitting an estimated $56 million in sales in the fourth quarter, the drugmaker said. That's not exactly blockbuster material yet, but the drug did post 20% quarter-to-quarter growth––a promising sign after slow initial uptake.

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Over the next couple years, Alnylam hopes to shepherd two of its meds to market alongside two partnered drugs with high expectations. The drugmaker has already submitted primary hyperoxaluria med lumarisan for FDA approval, and another ATTR amyloidosis candidate, vutrisiran, is currently enrolling phase 3 patients.

Meanwhile, partnered med inclisiran, a PCSK9 candidate recently bought out by Novartis, is facing an FDA approval decision this year, and a fourth med, hemophilia candidate fitusiran co-developed with Sanofi, is enrolling in phase 3.