March 30, 2021
As requested, and as part of our ongoing commitment to keep you informed of important milestones regarding Zolgensma® (onasemnogene abeparvovec), I am writing on behalf of Novartis to let you know that on Friday March 26th, the Canadian Agency for Drugs and Technologies in Health (CADTH) published their recommendation. Zolgensma has been recommended for reimbursement by public drug plans outside of Quebec for the treatment of pediatric patients who meet the following criteria:
- Genetic documentation of 5q SMA with biallelic mutations in the SMN1
- Symptomatic or pre-symptomatic with 1 to 3 copies of the SMN2
- Aged 180 days (6-months-old) or younger.
- Do not currently require permanent feeding or ventilatory support (either invasive or non-invasive).
While we welcome a positive recommendation, Novartis is disappointed that it is limited by age, without a mechanism for case-by-case review. The societal impact of unequal access due to delayed diagnoses for all SMA patients across Canada should have been a key consideration as we saw in the recommendation from the Institut national d’excellence en santé et services sociaux (INESSS), Quebec’s health technology assessment body.
Following Health Canada approval and the closure of the global managed access program to new enrollments in Canada, interim access for Zolgensma was put in place so that families affected by SMA would not be caught in limbo while we work through the formal reimbursement process. It’s troubling that families must continue to fight for treatment when all jurisdictions have the ability to provide Zolgensma now.
We anticipate the CADTH recommendation will be frustrating for the community, particularly as newborn screening for SMA has yet to be widely adopted across the country. Know that we will continue to advocate for the broadest possible access to Zolgensma, as we negotiate a final agreement for all Canadians with the pan-Canadian Pharmaceutical Alliance.
Vice President, Gene Therapy and Rare Diseases
Novartis Pharmaceuticals Canada Inc.