One step closer to achieving public reimbursement of AMVUTTRA^® for hATTR patients in Canada

MISSISSAUGA, ON, Oct. 18, 2024 /CNW/ - Alnylam Canada ULC is pleased to announce that it has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for the public reimbursement of AMVUTTRA® (vutrisiran injection), an RNAi therapeutic administered by quarterly subcutaneous injection, for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).¹ This comes following a positive recommendation from the Canadian Drug Agency (CDA) and the Institut national d'excellence en santé et services sociaux (INESSS), and signals the next step in the process where individual participating provinces and territories will start to list AMVUTTRA^® on their formularies.

"This is a major milestone in access for adults living with hATTR amyloidosis," said Colleen Coxson, Country General Manager, Alnylam Canada ULC. "Once product listing agreements are in place with all participating provinces and territories, nearly every Canadian patient diagnosed and living with this debilitating condition will have coverage for AMVUTTRA, which is a therapy that can quickly control the disease to avoid irreversible damage by rapidly knocking down² toxic TTR* in the body."

Hereditary ATTR amyloidosis is a rare disease which is inherited, progressively debilitating, and often fatal caused by mutations in the TTR gene.³ Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in peripheral sensory-motor neuropathy, autonomic neuropathy, and/or cardiomyopathy.³ hATTR amyloidosis represents a major unmet medical need, affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival (3.4 years) for patients with cardiomyopathy.³  The current standard of care is Onpattro (patisiran) which requires patients to receive several pre-medications before each 3.5-hour infusion every three weeks.⁴

"News of another funded treatment option for hATTR amyloidosis patients is a welcome development for those experiencing the debilitating effects of polyneuropathy," said Anne Marie Carr, Founder of the Hereditary Amyloidosis Canada (HAC). "As a patient myself, I understand the effects the disease can have, which is why I want to congratulate the pCPA, and Alnylam for reaching this important milestone so quickly.  Looking forward to the provincial governments quickly providing access to a treatment like AMVUTTRA, expanding treatment options can support improvements in quality of life for patients and their families."

The positive recommendations for reimbursement were supported by the results of the HELIOS-A Phase 3 study, a global, randomized, open-label, multicentre study to evaluate the efficacy and safety of AMVUTTRA in adult patients with hATTR amyloidosis. Based on the study, patients who received quarterly injections with AMVUTTRA significantly improved the signs and symptoms of hATTR amyloidosis, with nearly 50 per cent of patients experiencing an arrest or reversal of their polyneuropathy symptoms within 18 months.⁵

Full details on the recommendations are available on CDA and INESSS websites.

About AMVUTTRA^® (vutrisiran injection)²
AMVUTTRA is an RNAi therapy designed to silence a specific messenger RNA (mRNA) by blocking the production of the wild-type and variant transthyretin (TTR) protein before it is produced. AMVUTTRA utilizes Alnylam's GalNAc conjugate delivery platform and Enhanced Stabilization Chemistry (ESC), designed to increase potency and metabolic stability by allowing quarterly subcutaneous administration.

About HELIOS-A Phase 3 Study
HELIOS-A is a global, randomized, open-label, multicentre, Phase 3 study that evaluated the efficacy and safety of AMVUTTRA in a diverse group of hATTR patients with stage 1 or stage 2 polyneuropathy. The results of the HELIOS-A study were published in Amyloid in July 2022.

In the HELIOS-A study, the drug met the primary endpoint and all secondary endpoints of the study at both 9 months and 18 months. During the HELIOS-A 18-month treatment period, the most frequently occurring adverse reactions (≥10%) reported in patients treated with AMVUTTRA were pain in extremity (14.8%) and arthralgia (10.7%); none of the adverse reactions resulted in discontinuation of treatment. Vutrisiran demonstrated an improvement in the mean change from baseline in the modified neuropathy impairment score + 7 (mNIS+7) at 9 months (the primary endpoint), compared to external placebo data from the landmark APOLLO Phase 3 patisiran study.

*hATTR amyloidosis is caused by deposit of toxic misfolded TTR proteins.

About RNAi
RNAi (RNA interference) is a natural cellular gene silencing process that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a scientific breakthrough that occurs about once every ten years," and was recognized with the award of the 2006 Nobel Prize in Physiology or Medicine.

About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a new class of innovative medicines with the potential to transform the lives of people affected by rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful and clinically validated approach to the potential treatment of a wide range of serious and debilitating diseases. Founded in 2002, Alnylam is pursuing a bold vision to turn scientific possibilities into reality with a robust RNAi therapeutics platform. Alnylam has developed licensed RNAi therapeutics for the treatment of hATTR amyloidosis, acute hepatic porphyria, primary hyperoxaluria type 1 and primary hypercholesterolemia/mixed dyslipidemia. Alnylam has a broad pipeline of investigational drugs, including six product candidates that are in late-stage development. Alnylam is executing its "Alnylam P5x25" strategy to deliver transformative medicines in rare and common diseases that benefit patients worldwide through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. Alnylam Canada is headquartered in Mississauga, Ontario with established operations since June 2018.

AMV-CAN-00029 0924

SOURCE Alnylam Pharmaceuticals, Inc.

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