Alnylam Pharmaceuticals, Inc. has announced the U.S. Food and Drug Administration (FDA) approval of the supplemental new drug application (SNDA) for its RNAi therapeutic, Amvuttra® (vutrisiran). This approval expands the indication for Amvuttra, making it the first and only therapeutic approved by the FDA for the treatment of ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
The Helios-B Phase 3 clinical trial, which evaluated Amvuttra for the treatment of ATTR-CM, achieved statistical significance compared to placebo on all 10 pre-specified primary and secondary endpoints. In the overall population, Amvuttra reduced the risk of all-cause mortality (ACM) and recurrent cardiovascular (CV) events by 28% during the double-blind treatment period of up to 36 months.
Moreover, Amvuttra significantly reduced the risk of ACM and recurrent CV events by 33% in the double-blind period and significantly reduced the risk of mortality by 35% through 42 months in the monotherapy population. Patients treated with Amvuttra also experienced preservation of functional capacity and quality of life, as well as early improvements in biomarkers nt-proBNP and troponin I, which are predictive of cardiovascular outcomes.
In terms of safety and tolerability, the most common adverse reactions in patients treated with Amvuttra were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%). No new safety concerns were identified in the Helios-B clinical trial of patients with ATTR-CM.
The approval also signifies a beacon of hope for the amyloidosis community, with Amvuttra in hATTR-PN being covered by insurers for about 99% of patients, with the majority paying $0 out-of-pocket. Similar broad coverage and out-of-pocket costs are expected in ATTR-CM given comparable payer dynamics and the clinical value demonstrated in the Helios-B clinical trial.
Alnylam has a proven track record of working closely with payers to ensure broad patient access to its medicines, including pioneering innovative value-based agreements linked to clinical outcomes that ensure value and predictability for payers. The company offers multiple support services and resources for patients prescribed its products through Alnylam Assist®, designed to provide one-on-one support for patients and their healthcare teams to help navigate the treatment journey.
The marketing authorization applications based on Helios-B data are currently under review by several global health agencies, including the European Medicines Agency (EMA), the Brazilian Health Regulatory Agency (ANVISA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025. Today the company's shares have moved 11.8% to a price of $283.34. If you want to know more, read the company's complete 8-K report here.
