ONPATTRO® (patisiran) Now Reimbursed in Canada For the Treatment of Hereditary Transthyretin (TTR)-Mediated Amyloidosis (hATTR)

ONPATTRO is the only treatment that has demonstrated improvement in both polyneuropathy and quality of life measurements in patients with hATTR amyloidosis (relative to baseline)^{1, 2}

MISSISSAUGA, ON, Sept. 28, 2021 /CNW/ - Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) is pleased to announce that ONPATTRO^® (patisiran) is now reimbursed almost everywhere in Canada for the treatment of hereditary ATTR (hATTR) amyloidosis with polyneuropathy. This comes following a positive recommendation for reimbursement from the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en santé et services sociaux (INESSS).

"We are pleased to report that the vast majority of patients across Canada with provincial or private payer coverage now have access to ONPATTRO, and we are working to solidify access for patients in Prince Edward Island and Newfoundland as well," said Colleen Coxson, Country General Manager, Alnylam Pharmaceuticals. "This is a major milestone because ONPATTRO has the potential to reverse the polyneuropathy symptoms of the disease, which can lead to a better quality of life and better disease outcomes. The availability of ONPATTRO means that nearly every Canadian patient diagnosed with hATTR amyloidosis now has coverage for this life-changing therapy, giving new hope to them and their loved ones."

hATTR amyloidosis is a hereditary, multisystemic, progressive disease caused by mutations that interfere with the way the body manufactures a specific protein formed in the liver. Known as gene silencing, RNAi therapeutics is a new approach to the treatment of the disease; targeting the faulty protein that causes the condition. ONPATTRO is the only treatment that has demonstrated improvement, relative to baseline, in both polyneuropathy and quality of life measurements in patients with hATTR amyloidosis.^3,4 

"ONPATTRO offers a new option to patients suffering from hATTR amyloidosis, a very rare disease with limited therapeutic choices," said Dr. Nowell Fine, Cardiologist with Alberta Health Services, Clinical Director of the Libin Cardiovascular Institute, and Associate Professor of Cardiac Sciences at the University of Calgary. "The disease can be debilitating and is often fatal, with a median survival of just 4.7 years following diagnosis. With reimbursement almost everywhere in Canada, patients are one step closer to receiving a safe and effective treatment that has demonstrated improvements in both symptom management and quality of life for patients."

"Until recently, many patients suffering from hATTR did not have treatment for their condition, only symptom management," said Anne Marie Carr, Founder of the Hereditary Amyloidosis Canada (HAC).
"I want to congratulate the provinces on this encouraging milestone, reinforcing that Canadians with rare disease are top of mind. Accessibility to a treatment like ONPATTRO can provide life changing improvements to patients and their families."

The positive recommendations for reimbursement were supported by the results of the Phase III APOLLO study, which showed that ONPATTRO was associated with a statistically significant difference versus placebo in change from baseline, as well as improvements from baseline in key study end points for those receiving ONPATTRO versus placebo.⁵ Statistically significant differences in change from baseline versus placebo were also demonstrated favouring ONPATTRO for disability, gait speed, unintended weight loss, and autonomic symptoms.⁶ Full details on the recommendations are available on the CADTH and INESSS websites.

ONPATTRO uses innovative 'gene silencing' technology and is the first-ever RNAi therapeutic approved in Canada, laying the foundation for a new class of medicines in RNAi therapeutics.⁷ 

About ONPATTRO (patisiran)

Patisiran is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis with polyneuropathy. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. Patisiran blocks the production of transthyretin in the liver, reducing its accumulation in the body's tissues in order to halt or reverse the neurological progression of the disease.⁸ ONPATTRO recently won the 2021 Prix Galien Canada Award for Best Innovative Product as the first-ever Health Canada approved RNAi therapeutic.⁹ This marked ONPATTRO's fifth Prix Galien, having won the 2020 Best New Medicine Award in France and the 2019 and 2020 Prix Galien Award for Best Biotechnology Product in the United States, Italy, and Netherlands.

About hATTR amyloidosis¹⁰

Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. 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 intractable peripheral sensory-motor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis, represents a major unmet medical need with significant morbidity and mortality affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival (3.4 years) for patients presenting with cardiomyopathy.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents a promising and rapidly advancing frontier in biology and drug development that has the potential to transform the care of patients with genetic and other diseases. It was awarded the 2006 Nobel Prize for 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 improve the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS) diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform. Alnylam has a deep pipeline of investigational medicines, including five product candidates that are in Phase 3 clinical trials and one in registration. Looking forward, Alnylam will continue to execute on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam employs over 1,200 people worldwide and is headquartered in Cambridge, MA. Alnylam Canada is headquartered in Mississauga, Ontario with established operations since June 2018. 

SOURCE Alnylam Pharmaceuticals, Inc.