Onpattro Now Will Be Reimbursed for Most Patients in Canada

Marisa Wexler MS avatar

by Marisa Wexler MS |

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Onpattro (patisiran) is now reimbursed for patients with familial amyloid polyneuropathy (FAP) living across most regions of Canada, according to Alnylam Pharmaceuticals, the therapy’s developer.

The announcement 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), two agencies that help guide healthcare policy decisions in Canada.

“We are pleased to report that the vast majority of patients across Canada with provincial or private payer coverage now have access to Onpattro,” Colleen Coxson, country general manager at Alnylam, said in a press release.

“The availability of Onpattro means that nearly every Canadian patient diagnosed with [FAP] now has coverage for this life-changing therapy, giving new hope to them and their loved ones,” Coxson said, adding that Alnylam is now “working to solidify access for patients in Prince Edward Island and Newfoundland as well.”

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The recommendation will make the first-line therapy, given intravenously (into the vein), available for the first time to some people in Canada with FAP, according to Anne Marie Carr, founder of the Hereditary Amyloidosis Canada, a non-profit advocacy organization.

“Until recently, many patients suffering from [FAP] did not have treatment for their condition, only symptom management,” Carr said. “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.”

FAP is caused by mutations in the TTR gene, resulting in the production of a misfolded version of the protein transthyretin, which clumps together and builds up to toxic levels in different tissues and organs.

Onpattro uses a technology called RNA interference, or RNAi, to essentially stop the mutated gene from being used to create the misfolded protein, thereby lowering its levels in the body. Outside of Canada, Onpattro has been approved to treat adults with FAP in the U.S., Europe, Japan, Switzerland, and Brazil.  In the country, Onpattro is the first-ever Health Canada approved RNAi therapeutic.

Those approvals, as well as the positive recommendations for reimbursement from CADTH and INESSS, were supported by data from a Phase 3 clinical trial called APOLLO (NCT01960348).

The Alnylam-sponsored trial enrolled 225 FAP patients who were given either Onpattro or a placebo for 18 months. Results from the study showed that Onpattro was able to ease disease symptoms and improve patients’ quality of life.

“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,” said Nowell Fine, MD, a cardiologist with Alberta Health Services and associate professor at the University of Calgary.