The first approved RNAi therapy has made another breakthrough, and the Alnylam innovation platform has reported good news

< strong>▎WuXi Contex Content Team Editor

Today, Alnylam Pharmaceuticals, a star company in the RNAi field, announced that the first FDA-approvedThe RNAi therapy patisiran met the primary endpoint in a phase 3 clinical trial of transthyretin-mediated amyloidosis (ATTR-CM) patients with cardiomyopathy. Alnylam noted that the results are for the first time demonstrating the use of RNAi therapy to reduce transthyretin (TTR) expression, is an effective strategy for the treatment of ATTR amyloidosis with cardiomyopathy.

In this randomized, double-blind, placebo-controlled Phase 3 trial, a total of 360 Patients received patisiran or placebo. The results of the trial showed that patisiran met the primary endpoint of the trial. After 12 months of treatment, the 6-minute walking test results of patients receiving patisiran were significantly better than those of the control group compared with baseline (p=0.0162) strong>. In addition, the trial also met secondary endpoints of measures of health-related quality of life assessed using the KCCQ questionnaire. In the composite secondary endpoint of all-cause death and cardiovascular events, patisiran treatment did not show a statistically significant difference (p=0.0574).

Alnylam said detailed results of the study will be reported at a future medical meeting. Meanwhile, the company plans to submit a regulatory application to the U.S. FDA this year. If approved, patisiran is expected to significantly expand the ATTR amyloidosis patient population that could benefit. An article in the industry media Evalute pointed out that the first approved indications for patisiran included about 50,000 patients, and more than 300,000 patients with ATTR amyloidosis with cardiomyopathy.

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[1] Alnylam Reports Positive Topline Results from APOLLO -B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy. Retrieved August 3, 2022, from

[2] Topline Results from APOLLO -B Phase 3 Study of Patisiran. Retrieved August 3, 2022, from

[ 3] Alnylam’s heart beats stronger. Retrieved August 3, 2022, from

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