FAP therapy Amvuttra closer to reimbursement in Canada
Alnylam, pan-Canadian Pharmaceutical Alliance agree on reimbursement terms
Adults with familial amyloid polyneuropathy (FAP) living in Canada may soon be able to receive Amvuttra (vutisiran) at little or no cost through public drug plans, the therapy’s developer said.
Amvuttra is approved in the country for adult FAP patients with stage 1 or stage 2 polyneuropathy or mild to moderate symptoms of polyneuropathy, or damage to multiple nerves.
FAP is a form of hereditary transthyretin amyloidosis (hATTR amyloidosis) wherein mutations in the TTR gene result in the accumulation of toxic clumps of the transthyretin protein, mainly in the nerves outside the brain and spinal cord.
Alnylam Pharmaceuticals said Alnylam Canada has signed a letter of intent with the pan-Canadian Pharmaceutical Alliance (pCPA) for the public reimbursement of the injectable therapy. The letter essentially defines the terms and conditions under which participating publicly funded drug programs will reimburse a therapy.
The pCPA negotiated on behalf of those programs. Alnylam will now have to work with individual Canadian provinces and territories to have Amvuttra listed for coverage on specific drug plans.
‘Major milestone’
“This is a major milestone in access for adults living with hATTR amyloidosis,” Colleen Coxson, country general manager of Alnylam Canada, said in a company press release. “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.”
The agreement followed positive recommendations for public reimbursement of the therapy from the Canadian Drug Agency and the Institut national d’excellence en santé et services sociaux (INESSS).
Progress toward Amvuttra’s public reimbursement was met with enthusiasm from the patient community.
“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, the founder of Hereditary Amyloidosis Canada. “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.”
Amvuttra is designed to reduce production of the faulty transthyretin from its genetic template as a way of preventing further nerve damage. Its mechanism of action is similar to that of Alnylam’s Onpattro (patisiran), but while the older FAP therapy is given via infusions into the bloodstream once every three weeks, Amvuttra is administered via under-the-skin injections once every three months.
The next-generation therapy is also approved in Europe for the same indication and in the U.S. for adults with FAP.
Data supporting Amvuttra’s regulatory approvals and the more recent positive recommendations for its reimbursement in Canada came from the Phase 3 HELIOS-A clinical trial (NCT03759379). Results showed that the treatment led to significant reductions in polyneuropathy symptoms as well as improvements in life quality, walking speed, functional abilities, and nutritional status among adults with FAP.
“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,” Carr said.
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