First-ever FDA approval for an RNA interference drug granted to Alnylam's costly Onpattro treatment for hATTR-caused nerve damage

November 02, 2018 | Case Study

Alnylam Pharmaceuticals is a relatively small Boston-based company, founded in 2002, that has been burning through cash – last year, it spent more than $0.5 billion and made only about $100 million. Its lead product, revusiran, failed in clinical trials in 2016, but Alnylam has nonetheless kept up some momentum, including interest from pharmaceutical giant Sanofi (the company bought a minority share of Alnylam), as well as an acquisition of one of Merck's subsidiaries, Sirna Therapeutics. In mid-2018, Alnylam's efforts were rewarded by the U.S. FDA, with Alnylam's Onpattro drug receiving the first-ever RNA interference drug approval. Onpattro is aimed at patients suffering from hereditary transthyretin-mediated amyloidosis (hATTR), a rare neurodegenerative brain disease affecting about 50,000 people worldwide.

About Lux Research

Lux Research is a leading provider of tech-enabled research and advisory solutions, helping clients drive growth through technology innovation. A pioneer in the research industry, Lux uniquely combines technical expertise and business insights with a proprietary intelligence platform, using advanced analytics and data science to surface true leading indicators. With quality data derived from primary research, fact-based analysis, and opinions that challenge traditional thinking, Lux clients are empowered to make more informed decisions today to ensure future success.

Already a Lux Client?

Sign In

Interested in Learning More?

Contact us to learn the benefits of becoming a Lux member.

Or call us now

For North America (Boston Headquarters)
+1 (617) 502-5300

For EMEA (Amsterdam)
+31 20 280 7900

For APAC (Singapore)
+65 6592-6978