Kye Pharmaceuticals Announces Milestone of CDAand INESSS Reimbursement Recommendations for AGAMREE™ (vamorolone), the First Therapy Approved in Canada for Duchenne Muscular Dystrophy
Canada’s Drug Agency (CDA) and the Institut national d’excellence en santé et en services sociaux (INESSS) recommendations represent an important milestone toward access to AGAMREETM for Canadians living with Duchenne muscular dystrophy
Mississauga,Ontario – September 28th, 2026 – Kye Pharmaceuticals welcomes the recent Health Technology Assessment (HTA) final recommendations issued by CDA and INESSS for AGAMREE (vamorolone), the first therapy approved in Canada for Duchenne muscular dystrophy.
CDA Issues Positive Reimbursement Recommendation1
CDA, through the Canadian Drug Expert Committee (CDEC), issued a positive reimbursement recommendation for AGAMREE for pediatric patients with Duchenne muscular dystrophy. Kye contends that the final recommendation from CDA fails to acknowledge the differentiated value of vamorolone vs prednisone via mitigation of serious side effects, potentially complicating the viability of equitable access to vamorolone for patients in Canada.
In February 2026, CDA issued a draft recommendation2 for vamorolone, electing not to acknowledge a differentiation on side effects (i.e. reduction of harms) compared to prednisone and citing the need for longer term data to reduce uncertainty. Based on feedback from clinicians and the Duchenne community, Kye filed a request for reconsideration with CDA to include emerging longer-term data from upcoming publications characterizing the reduction of specific side effects, differentiating AGAMREE from classic corticosteroids. Although the reconsideration was accepted by CDA the key differentiating side effects including bone health, height and eye health were not reflected in the final recommendation. The benefits versus classic corticosteroids have been observed firsthand in Canada with patients participating in clinical trial programs for over 8 years.
INESSSHighlights Therapeutic Value of AGAMREETM3
INESSS acknowledged the therapeutic value of AGAMREE for the treatment of DMD. While recognizing the effectiveness of classic corticosteroids in slowing disease progression, they emphasize the unfavorable safety profile of these treatments. Consequently, INESSS acknowledged the significant unmet health need for people with DMD to slow disease progression, prolong survival, and improve quality of life and autonomy.
Compared to prednisone, the committee highlighted improvements in the safety profile including fewer growth-stunting effects, reduced behavioural issues, and positive clinical trial results indicating that vamorolone may reduce vertebral fractures. However, despite the positive evidence, INESSS did not recommend listing AGAMREE at this time due to economic factors.
Together, these assessments recognize the therapeutic value of AGAMREE in treating Duchenne and reinforce the importance of timely equitable access for Canadians living with this rare disease.
“We appreciate that both CDA and INESSS acknowledge the therapeutic value of AGAMREE and the potential benefits it can provide to individuals living with Duchenne.” said John McKendry, President of Kye Pharmaceuticals. “We are grateful to the dozens of Canadian patients and researchers who participated in the clinical trial programs, generating the data that has enabled this medication to be authorized for sale by Health Canada as the first approved treatment for DMD. Moving forward, our collective focus will be to secure the appropriate provincial and federal formulary listings so that all patients with DMD would have timely access to treatment."
About AGAMREETM (vamorolone)
AGAMREE's unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid that has the potential to demonstrate comparable efficacy to steroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow AGAMREETM to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD. In the pivotal VISION-DMD study, AGAMREETM met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. Adverse events were generally of mild to moderate severity.4
About Kye Pharmaceuticals
Kye is a leading Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of innovative medicines, Kye’s portfolio spans a range of therapeutic areas including cardiology, psychiatry, pediatrics, rare diseases, neuromuscular, hematology, and neurology. Kye Pharmaceuticals is a private company, founded in Canada and focused exclusively on the healthcare needs of patients in Canada. Kye aims to provide medicines that deliver better outcomes to our partners, Canadian healthcare professionals, and most importantly, patients across Canada. Kye, headquartered in Mississauga, Ontario was recognized on the Globe & Mail Canada’s Top Growing Companies list for 2025 and 2026.
Media and InvestorContact:
John McKendry, CEO, Kye Pharmaceuticals
1-888-822-7126
johnm@kyepharma.com
Denise David, VP Scientific Affairs, Kye Pharmaceuticals
1-888-822-7126
References:
1Canada’s Drug Agency. Vamorolone: reimbursement recommendation [Internet]. Ottawa(ON): Canada’s Drug Agency; 2026 [cited 2026 Sep 25]. Available from:https://www.cda-amc.ca/vamorolone
2Canada’s Drug Agency. Vamorolone: reimbursement recommendation [Internet]. Ottawa(ON): Canada’s Drug Agency; 2026 [cited 2026 Feb 19]. Available from: https://www.cda-amc.ca/vamorolone
3Institut national d’excellence en santé eten services sociaux. Agamree (dystrophie musculaire de Duchenne)[Internet]. Québec (QC): INESSS [cited 2026 April 29]. Available from: INESSS: Médicament: Agamree (dystrophiemusculaire de Duchenne)
4Dang UJ et al. (2024) Neurology 024;102:e208112. doi.org/10.1212/WNL.0000000000208112
