10 August 2018 - This morning, after 16 years and $2.5 billion in investment, the Cambridge, Massachusetts-based company Alnylam finally turned a 1998 biology breakthrough into a medicine: a drug called Onpattro to treat the destruction of nerves that results from a rare disease, hATTR amyloidosis, thought to afflict about 50,000 people worldwide.

“It’s a great day for patients, it’s a great day for science, and, frankly it's a great day for perseverance,” says John Maraganore, Alnylam’s longtime chief executive.

The price of the new medicine, though, may give people who haven’t paid attention to the cost of treatments for rare diseases pause. Onpattro will have an list price of $450,000 per patient per year for the average patient. (The drug is dosed based on weight.) This is in line with other drugs that treat rare disorders and the expectations of financial analysts. In order to make sure patients can get it, Alnylam is taking steps that are new for a rare-disease company, including a money-back guarantee for insurers.

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