Roche Canada and the Pan-Canadian Pharmaceutical Alliance (pCPA) Successfully Complete Negotiations for EVRYSDIⓇ (Risdiplam) for the Treatment of Adults and Children With Spinal Muscular Atrophy (SMA)
MISSISSAUGA, ON, Feb. 3, 2022 /CNW/ - Hoffmann-La Roche Limited (Roche Canada) today announced that it has successfully completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) for EVRYSDI® (risdiplam), a pre-mRNA splicing modifier of survival of motor neuron 2 (SMN2) designed to treat SMA, indicated for the treatment of spinal muscular atrophy (SMA) in patients two months of age or older.1
"Cure SMA Canada is delighted that EVRYSDI has moved one step closer to being accessible to Canadian patients," said Susi Vander Wyk, Executive Director, Cure SMA Canada. "As a community, we look forward to the benefit of available treatment options, and meeting the needs for a wide range of SMA patients. We encourage the provincial payers to recognize the treatment gap and offer a path for those still waiting."
Shortly following the successful completion of pCPA negotiations, Quebec is the first province to fund EVRYSDI. The Quebec Minister of Health and Social Services has added EVRYSDI to the Liste des médicaments and the Liste des médicaments - Établissements effective February 2, 2022. The specific criteria for reimbursement are now available on the Régie de l'assurance maladie du Québec (RAMQ) website. This listing follows positive recommendations by INESSS for EVRYSDI issued in 2021.
"I am pleased to see such positive news for the SMA community," said Dr. Xavier Rodrigue, Physiatrist at the l'Institut de Réadaptation en Déficience Physique de Québec (IRDPQ). "The successful completion of negotiations represents another step towards access to another treatment option that can improve quality of life for Canadians with SMA."
As a next step, Roche Canada will continue working with other provincial jurisdictions to make EVRYSDI available as soon as possible through public drug plans.
About EVRYSDIⓇ (risdiplam)
EVRYSDI is a pre-mRNA splicing modifier of survival of motor neuron 2 (SMN2) designed to treat SMA, which is caused by mutations in chromosome 5q that lead to SMN protein deficiency. EVRYSDI works by helping the body make more of a protein called, "SMN protein". EVRYSDI increases and sustains the amount of SMN protein in the body, which helps to treat SMA. EVRYSDI is a strawberry-flavoured liquid taken once daily by mouth or feeding tube.1
About Spinal Muscular Atrophy (SMA) SMA is a progressive neuromuscular condition that affects the nerve cells in the spinal cord and impacts the muscles used for activities such as breathing, eating, crawling and walking.2,3 It affects approximately one in 6,000 babies born, and about one in 40 people are genetic carriers.2 SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.4
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1 EVRYSDI Product Monograph, April 14, 2021.
2 Cure SMA, « About SMA », accessible à l'adresse https://www.curesma.org/about-sma/. Dernière consultation le 20 janvier 2022.
3 Treat-NMD Neuromuscular Network, Disease Information- Spinal Muscular Atrophy, accessible à l'adresse https://treat-nmd.org/treat-nmd-diseases/spinal-muscular-atrophy/. Dernière consultation le 20 janvier 2022.
4 Anderton RS, Mastaglia FL. Advances and challenges in developing a therapy for spinal muscular atrophy. Expert Rev Neurother. 2015;15(8):895-908. doi: 10.1586/14737175.2015.1059757. PMID: 26200127. Last accessed January 20, 2022.