Alnylam Submits Type II Variation to EMA for Vutrisiran in ATTR Amyloidosis with Cardiomyopathy
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Oct. 16, 2024—Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced the submission of a Type II Variation to the European Medicines Agency (EMA) for vutrisiran, an RNA interference (RNAi) therapeutic aimed at treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). Vutrisiran, also known as AMVUTTRA®, is already approved in the EU for hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy.
“Today marks a significant milestone in our mission to provide RNAi therapeutics to patients in need,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “ATTR-CM is a life-threatening condition that causes heart failure. Vutrisiran rapidly reduces TTR levels, and results from the HELIOS-B study show it significantly lowers all-cause mortality and cardiovascular events.”
This regulatory application is based on positive outcomes from the HELIOS-B Phase 3 study, which met all ten primary and secondary endpoints with statistical significance. Vutrisiran improved mortality rates, cardiovascular events, functional capacity, quality of life, and heart failure symptoms. The safety profile was consistent with that seen in hATTR amyloidosis treatments, with adverse events occurring similarly in both vutrisiran and placebo groups. Detailed findings from HELIOS-B were published in The New England Journal of Medicine.
Additionally, a supplemental New Drug Application (sNDA) for vutrisiran has been submitted to the U.S. FDA for ATTR-CM treatment, with further global submissions planned.
AMVUTTRA® (vutrisiran) Indication and Important Safety Information
Indication: Vutrisiran is indicated in Europe and the UK for treating hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
Important Safety Information: Vutrisiran can lower serum vitamin A levels; supplementation of 2,500 to 3,000 IU per day is recommended. Patients should be referred to an ophthalmologist if they exhibit ocular symptoms related to vitamin A deficiency.
Adverse Reactions: Common adverse reactions include pain in extremities, arthralgia, dyspnoea, injection site reactions, and increased blood alkaline phosphatase levels.
For additional information about vutrisiran, refer to the full Summary of Product Characteristics.
About AMVUTTRA® (vutrisiran)
Vutrisiran is an RNAi therapeutic that targets both variant and wild-type transthyretin (TTR), addressing the root cause of ATTR amyloidosis. Administered quarterly via subcutaneous injection, it is approved in over 15 countries for treating hATTR amyloidosis polyneuropathy (hATTR-PN) in adults and is also being developed for ATTR-CM, including wild-type and hereditary forms of the disease.
About ATTR
Transthyretin amyloidosis (ATTR) is a rapidly progressive and often fatal disease caused by misfolded TTR proteins that accumulate in various body parts, including the heart and nerves. There are two forms of ATTR: hereditary (hATTR) and wild-type (wtATTR), impacting approximately 50,000 and 200,000-300,000 people globally, respectively.
About RNAi
RNA interference (RNAi) is a natural gene-silencing process that has led to the development of RNAi therapeutics, a groundbreaking class of medicines. By targeting messenger RNA (mRNA), these therapeutics can prevent the production of disease-causing proteins, representing a transformative approach to treating genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is a leader in RNAi therapeutics, focused on developing innovative medicines for rare and prevalent diseases with unmet needs. Founded in 2002, Alnylam continues to advance its “Alnylam P5x25” strategy to deliver transformative therapies and sustainable innovation, aiming to improve the lives of patients globally. The company is headquartered in Cambridge, MA.
