Alnylam Receives Positive Recommendation From Canada's Drug Agency (CDA) for the Public Reimbursement of AMVUTTRA® (vutrisiran injection), the First and Only RNAi Therapeutic for the Treatment of Cardiomyopathy in Adult Patients with ATTR Amyloidosis
MISSISSAUGA, ON, March 2, 2026 /CNW/ - Alnylam Canada ULC is pleased to announce it has received a positive recommendation from Canada's Drug Agency (CDA) for the public reimbursement of its RNAi therapeutic, AMVUTTRA®.1 In December 2025, AMVUTTRA® was authorized by Health Canada for the treatment of cardiomyopathy in adult patients with wild-type or hereditary transthyretin-mediated amyloidosis (wtATTR or hATTR amyloidosis). This is an expanded indication for AMVUTTRA®, which was originally authorized for sale in Canada in 2023 for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hATTR amyloidosis.2
Cardiomyopathy associated with wild-type or hereditary transthyretin amyloidosis (ATTR-CM) is a serious, progressive, and ultimately fatal disease.3,4 There is no cure for ATTR-CM, which is caused by misfolded transthyretin (TTR) fibrils (malformed proteins) that build up primarily in your heart and elsewhere in the body. This makes it harder for the heart to pump blood, which can cause irreversible damage, and lead to heart failure.5 Patients' life expectancy after diagnosis is currently two to five years, underscoring the critical need for early and accurate detection to slow disease progression.6
"This recommendation brings us one step closer to delivering access to a first‑in‑class treatment that has a fundamentally different mechanism of action, targeting the root cause of the disease, rather than simply managing the symptoms," says Colleen Coxson, Country General Manager, Alnylam Canada ULC. "AMVUTTRA® expands the treatment landscape, offering patients and physicians an important new option that helps preserve function, enhance quality of life, and support longer survival. Our hope is that patients will gain swift access to this much needed therapy, and we remain committed to collaborating with all relevant partners to help make this a reality."
The positive recommendation for reimbursement was supported by the results of the HELIOS-B Phase 3 study, a randomized, double-blind, placebo-controlled, multicenter, global trial that enrolled adult patients with wtATTR or hATTR amyloidosis with cardiomyopathy. Detailed results from the HELIOS-B study were published in the The New England Journal of Medicine.
AMVUTTRA® is an RNAi therapeutic that specifically targets variant and wild-type TTR messenger RNA.7 By interfering with TTR production, AMVUTTRA® substantially decreases deposition of misfolded TTR fibrils, which form amyloid deposits and cause irreversible cardiovascular damage and premature death in patients with ATTR-CM.8
About AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) suppresses variant and wild-type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered via subcutaneous injection once every three months, AMVUTTRA® is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. It has been approved in the EU, Japan, the United States (U.S.), Canada, Brazil, United Arab Emirates (UAE), United Kingdon (UK) and Switzerland for the treatment of the cardiomyopathy of wild-type or hereditary ATTR amyloidosis in adults.
About the HELIOS-B Study9
In the HELIOS-B study, AMVUTTRA® met all 10 pre-specified primary and secondary endpoints across both the overall and monotherapy populations. These included statistically significant reductions in all-cause mortality and recurrent cardiovascular events, as well as significant improvements in functional capacity (6-minute walk test), health status and quality of life (Kansas City Cardiomyopathy Questionnaire), and heart failure symptoms and severity (NYHA class). The safety profile of AMVUTTRA® in HELIOS-B was consistent across all subgroups studied including age, sex, race, geographic region, type of ATTR amyloidosis (hATTR or wtATTR), baseline tafamidis use, and NYHA class.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today.10 Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was awarded the 2006 Nobel Prize for Physiology or Medicine.11 By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing or disease pathway proteins – thus preventing them from being made.12 This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health. Alnylam is headquartered in Cambridge, MA. Alnylam Canada ULC is headquartered in Mississauga, Ontario with established operations since June 2018.
AMV-CAN-00119 Feb 2026
1 https://www.cda-amc.ca/vutrisiran-0
2 AMVUTTRA® Product Monograph. Alnylam Netherlands B.V. Dec. 12, 2025
3 Mathew S. Maurer, MD, Sabahat Bokhari, MD, Thibaud Damy, MD, PhD, Sharmila Dorbala, MD, Brian M. Drachman, MD, Marianna Fontana, PhD, Martha Grogan, MD, Arnt V. Kristen, MD, Isabelle Lousada, MA, Jose Nativi-Nicolau, MD, Candida Cristina Quarta, MD, PhD, Claudio Rapezzi, MD, Frederick L. Ruberg, MD, Ronald Witteles, MD, and Giampaolo Merlini, MD. Expert consensus recommendations for the suspicion and diagnosis of transthyretin cardiac amyloidosis. Available at: https://www.ahajournals.org/doi/full/10.1161/CIRCHEARTFAILURE.119.006075
4 Mohamed-Salem, L., Santos-Mateo, J. J., Sanchez-Serna, J., Hernández-Vicente, Á., Reyes-Marle, R., Castellón Sánchez, M. I., Claver-Valderas, M. A., Gonzalez-Vioque, E., Haro-Del Moral, F. J., García-Pavía, P., & Pascual-Figal, D. A. (2018). Prevalence of wild type ATTR assessed as myocardial uptake in bone scan in the elderly population. International Journal of Cardiology, 270, 192–196.
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6 Nativi-Nicolau, J. N., Karam, C., Khella, S., & Maurer, M. S. (2022). Screening for ATTR amyloidosis in the clinic: overlapping disorders, misdiagnosis, and multiorgan awareness. Heart failure reviews, 27(3), 785–793. https://doi.org/10.1007/s10741-021-10080-2
7 AMVUTTRA® Product Monograph. Alnylam Netherlands B.V. Dec. 12, 2025
8 AMVUTTRA® Product Monograph. Alnylam Netherlands B.V. Dec. 12, 2025
9 AMVUTTRA® Product Monograph. Alnylam Netherlands B.V. Dec. 12, 2025
10 Elbashir, S. M., Harborth, J., Lendeckel, W., Yalcin, A., Weber, K., & Tuschl, T. (2001). Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature, 411(6836), 494–498. https://doi.org/10.1038/35078107
11 Zamore P. D. (2006). RNA interference: big applause for silencing in Stockholm. Cell, 127(6), 1083–1086. https://doi.org/10.1016/j.cell.2006.12.001
12 Elbashir, S. M., Harborth, J., Lendeckel, W., Yalcin, A., Weber, K., & Tuschl, T. (2001). Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature, 411(6836), 494–498. https://doi.org/10.1038/35078107
SOURCE Alnylam Pharmaceuticals, Inc.
Media Contacts: Proof Strategies, Alison O'Mahony, 416-912-8138
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