Novartis Applauds Quebec’s Move to Reimburse Families for Zolgensma

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by Yedida Y Bogachkov PhD |

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Novartis Pharmaceuticals Canada is lauding the province of Quebec for its decision to provide public reimbursement for Zolgensma (onasemnogene abeparvovec), an approved gene therapy for spinal muscular atrophy (SMA) in children.

Zolgensma is the first gene therapy to receive formal public reimbursement in Canada. The current recommendation follows that of the Institut national d’excellence en santé et services sociaux (INESSS) made earlier this year, which includes a pathway for children up to six months old to gain access to the medication, and for case-by-case access to Zolgensma for children older than six months.

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The treatment and criteria for reimbursement will be added soon to the list of medications on the Régie de l’assurance maladie du Québec website.

“This is a momentous day and one we know the Quebec SMA community has been waiting for,” Andrea Marazzi, Country Pharma Organization Head at Novartis Pharmaceuticals Canada, said in a press release. “I want to thank the Quebec government for their leadership, both in moving quickly to cover Zolgensma, as well as adopting the INESSS recommendations, which acknowledge the societal impact of unequal access due to delayed diagnoses for Quebec children with SMA.”

This decision comes after the Canadian Agency for Drugs and Technologies in Health announced it would not recommend reimbursement for Zolgensma in children with SMA who are more than 6 months old, citing insufficient evidence of efficacy and safety in older patients. However, the agency has recommended reimbursement for children up to 6 months of age.

“We believe all Canadian children with SMA who may benefit from this treatment should have the same opportunity and will continue to collaborate with the provinces, territories and federal plans to provide timely and equitable public reimbursement,” Marazzi said.

Zolgensma is a one-time gene therapy designed to replace the missing or defective SMN1 gene, the main cause underlying SMA. It is approved in the U.S. and Japan for patients under the age of 2, and conditionally approved in Europe for patients who weigh 21 kg (about 46 lbs) or less, with type 1 SMA or with three or fewer copies of the backup SMN2 gene.

The therapy was also approved in Canada to treat pediatric SMA patients who have three or fewer copies of the SMN2 gene, or with infantile-onset disease.

“Prompt access to treatments like Zolgensma that can halt progression of SMA are critical for children to reach their developmental potential and having costs covered by the province helps remove some of the impact that SMA can have on families,” said Nicolas Chrestian, chief of pediatric neurology at Centre Hospitalier Mère Enfant Soleil, Université Laval, in Québec City.

SMA affects one in 10,000 live births every year in Canada.