Health Canada has approved AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older, making it the first approved therapy for this devastating neuromuscular disease in Canada. The approval, granted under Health Canada's Priority Review process, represents a significant milestone for the more than 800 boys and young men living with DMD across Canada.
Novel Mechanism Offers Alternative to Standard Care
AGAMREE is a dissociative anti-inflammatory drug with a unique mode of action that binds to the same receptor as glucocorticoids but modifies downstream activity differently. Unlike traditional corticosteroids, AGAMREE is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues.
This mechanism has demonstrated the potential to 'dissociate' efficacy from steroid safety concerns, positioning AGAMREE as an alternative to existing corticosteroids, which are the current standard of care for children and adolescent patients with DMD.
Pivotal Trial Results Support Approval
The Health Canada approval was based on data from the pivotal Phase 2b VISION-DMD study, supplemented with safety information from four open-label studies including extension studies. In the VISION-DMD trial, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo with statistical significance (p=0.002) at 24 weeks of treatment.
The drug was administered at doses ranging from 2 to 6 mg/kg/day for periods extending up to 48 months. Compared with current standard of care corticosteroids, this novel treatment exhibited comparable efficacy while demonstrating data suggesting a reduction in adverse events, particularly related to bone health, growth trajectory, and behavior.
Improved Safety Profile
Currently available data show that AGAMREE, unlike traditional corticosteroids, has no restriction of growth and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers. The most commonly reported side effects in the VISION-DMD study were cushingoid features, vomiting, weight increase, and irritability, with adverse events generally of mild to moderate severity.
"Given the significant unmet need of individuals with DMD, I am pleased that AGAMREE® received priority review and is now approved by Health Canada," said Dr. Jean Mah, Director of the Pediatric Neuromuscular Program at the Alberta Children's Hospital in Calgary. "As AGAMREE® has no impact on linear growth and showed favorable changes in serum bone turnover biomarkers, it provides families with a viable treatment option compared to other corticosteroids."
Commercial Partnership and Access
Kye Pharmaceuticals holds exclusive Canadian commercial rights to AGAMREE for DMD treatment following a sub-license agreement with Catalyst Pharmaceuticals, Inc. in July 2024. Santhera Pharmaceuticals, which holds the original license from ReveraGen, will receive royalties on Canadian sales.
"This Health Canada approval reflects Kye Pharmaceuticals' commitment to advancing treatments for rare diseases and serving the unmet needs of Canadian patients," stated John McKendry, President and CEO at Kye Pharmaceuticals. "We remain committed to working alongside the DMD community and with provincial drug plans and private insurers across Canada to ensure AGAMREE® is accessible to individuals living with DMD."
Community Support and Research Investment
The approval was welcomed by Canadian DMD patient organizations, including Defeat Duchenne Canada, which helped fund one of the open-label studies for AGAMREE. This Canadian data, which included boys from five different trial sites across Canada in age ranges often neglected in Duchenne clinical trials, supported the Health Canada approval and broad indication.
"It's very impactful knowing that the first medicine approved in Canada is one that Defeat Duchenne Canada supported through research," said John Davidson, Founder of Jesse's Journey and current board member for Defeat Duchenne Canada.
Global Regulatory Success
This Canadian approval represents the sixth independent approval by a local health authority, following positive regulatory decisions in the U.S., Europe, the UK, China, and Hong Kong. The global regulatory success underscores the clinical significance of AGAMREE as a treatment option for DMD patients worldwide.
DMD is a rare inherited X-chromosome-linked disease that almost exclusively affects males, characterized by inflammation present at birth or shortly thereafter. The disease leads to progressive muscle degeneration and weakness, with major milestones including loss of ambulation, loss of self-feeding, the start of assisted ventilation, and development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure.
