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Alnylam estimates between 30,000 to 50,000 patients in the U.S. Onpattro is already approved for a form of transthyretin amyloidosis that affects nerves, a condition known as polyneuropathy for which it’s the top-selling medicine. But the company lost $853 million in 2021 and another $240 million in the first three months of this year.Īnalysts and investors view transthyretin amyloidosis with cardiomyopathy as a major opportunity for expanding Onpattro’s use and for Alnylam to reach consistent profitability. The company has climbed industry ranks over the past two decades to become one of the most productive biotechs, winning approval of four rare disease drugs and discovering a cholesterol-lowering medicine now sold by Novartis. Still, the positive primary results are likely to boost Alnylam, a trailblazer in the drugmaking method known as RNA interference. Infusion-related reactions, joint stiffness and muscle spasms occurred more frequently among those on Onpattro versus those receiving placebo.Īs Onpattro did not result in significant improvement on the secondary composite measure, formal statistical comparisons weren’t conducted on the two remaining secondary goals. Rates of side effects and serious adverse reactions were similar between the two groups. “ frankly validate the hypothesis that we’ve been working towards for a while.”īased on the results, Alnylam plans to apply later this year for an expanded Food and Drug Administration approval to treat the heart condition, called transthyretin amyloidosis with cardiomyopathy.įive patients taking Alnylam’s drug died while on the study, compared to eight patients on placebo. “We do think these effects are really clinically significant,” said Pushkal Garg, Alnylam’s chief medical officer, in an interview conducted under embargo, before the results were made public.

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Full results will be disclosed at a medical meeting in early September, Alnylam said in its Thursday statement announcing the trial results.

However, treatment did not lead to a statistically significant difference in a composite assessment, which tracked deaths from any cause and cardiovascular events as well as change in walk test scores.Īlnylam did not report detailed data, leaving the magnitude of the drug's benefits unclear. Patients on Alnylam’s medicine also reported improved quality of life on a self-administered questionnaire compared to the placebo group, meeting a secondary goal of the trial.

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