Alnylam wins FDA approval of rare disease drug in step toward profitability

Dive Brief:

• Alnylam Pharmaceuticals on Monday said the Food and Drug Administration has approved a new drug it’s been developing for the rare genetic disease transthyretin amyloidosis and that’s viewed by analysts as important to the company’s goal of becoming sustainably profitable.
• The drug, which will be sold as Amvuttra with an annual list price of $463,500, is cleared to treat the neurological symptoms, or polyneuropathy, associated with the inherited form of the disease. In clinical testing, Amvuttra stopped or reversed signs of the disease in more than half of treated patients after nine months.
• Amvuttra is the fifth Alnylam medicine approved since 2018, It’s meant to be a longer-lasting and more convenient version of the company’s top-selling drug, Onpattro, which earned $475 million in sales last year. Like Onpattro, the drug is also in late-stage testing for TTR cardiomyopathy, a more common form of the disease.

Dive Insight:

Alnylam, a pioneer in the drugmaking method known as RNA interference, has climbed the biotech industry ranks over the last two decades. After solving challenges associated with delivering these treatments into the body, Alnylam has become one of the sector’s more productive companies, winning approvals of four drugs for rare diseases and discovering a cholesterol-lowering medicine sold by Novartis.

Alnylam still isn’t profitable, however. The company lost $853 million in 2021 and another $240 million in the first quarter this year. Losses are expected to continue in the near future, too, even as Alnylam expects product revenue to climb as high as $930 million in 2022.

Amvuttra could help change that. The drug is more convenient to take than Onpattro, administered every three months as a subcutaneous injection rather than via infusion every three weeks. Patients also don’t need to take steroids to prepare for treatment, as they do with Onpattro. The two drugs have the same annual list price, Chief Commercial Officer Tonga Tanguler said on a conference call. 

Amvuttra might also help Alnylam stay ahead of a group of rivals that includes Ionis Pharmaceuticals, AstraZeneca and Intellia Therapeutics. Ionis already has a TTR amyloidosis drug on the market and a next-generation version in late-stage testing, while Intellia’s gene editing drug has shown promise in an early study.

Wall Street analysts have high expectations for Amvuttra. In an interview earlier this year, Stifel analyst Paul Matteis said the medicine and Onpattro could become a “Soliris-like drug franchise” for Alnylam, referring to the rare disease treatments Alexion Pharmaceuticals turned into a multibillion dollar business.

But to get there, Alnylam will likely need to succeed in TTR cardiomyopathy, which is thought to affect 10 times more people than the roughly 30,000 to 50,000 in the U.S. and Europe who have polyneuropathy.

Results are expected by mid-year from a Phase 3 trial of Onpattro known as Apollo-B. The study is a polarizing subject among biotech investors and a proxy for Amvuttra, for which a late-stage study should produce findings in 2024, according to a federal clinical trials database.

If Amvuttra were to succeed in that study, revenue between $3 billion to $5 billion is “seen as plausible,” Matteis said earlier this year.

The drug will be available in the coming weeks, and its first commercial sales are expected in early July, according to Tanguler.

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