Health Canada Approves AGAMREE® (vamorolone) as the First Treatment for Duchenne Muscular Dystrophy in Canada

MISSISSAUGA, ON, Oct. 2, 2025 /PRNewswire/ - Kye Pharmaceuticals ("Kye") announced today that Health Canada has approved AGAMREE^® (vamorolone) for the treatment of Duchenne Muscular Dystrophy (DMD) in boys age 4 and older.¹ This approval – granted under Health Canada's Priority Review process – marks an important milestone making AGAMREE ^® the first treatment in Canada approved for DMD a muscle wasting disease that affects predominantly young boys. DMD is a rare, debilitating and life-shortening neuromuscular disease.² The Canadian Neuromuscular Disease registry estimates that there are more than 800 boys and young men living with DMD in Canada and coping with its debilitating symptoms.³ DMD is a type of muscular dystrophy that causes weakness and a wasting of the muscle due to the absence of a protein called dystrophin. If the body is lacking dystrophin, muscle cells become damaged and movements that are considered natural such as getting up from the floor and walking become challenging. Boys with DMD typically lose the ability to walk in their early teens and have increasing cardiac and respiratory difficulties. Bone thinning and scoliosis are also common for those with Duchenne.²

Corticosteroids are the standard of care for DMD and although efficacious in terms of extending the ability for these boys to walk for a few more years, the side effects of weight gain, growth stunting, fractures, and behaviour, among others, often prevent them from being able to tolerate the recommended dose of these medications.⁴

"Given the significant unmet need of individuals with DMD, I am pleased that AGAMREE^® received priority review and is now approved by Health Canada. 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," says Dr. Jean Mah, Director of the Pediatric Neuromuscular Program at the Alberta Children's Hospital in Calgary.

The Health Canada approval of AGAMREE^® was based on the data from the pivotal Phase 2b VISION-DMD study as supplemented with safety information collected from four open-label studies, including extension studies. In these trials, AGAMREE^® was administered at doses ranging from 2 to 6 mg/kg/day, extending for a period of up to 48 months. Compared with current standard of care corticosteroids, this novel corticosteroid treatment exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory and behavior.⁵

AGAMREE^® is approved in the US, EU, UK, and China. AGAMREE^® was licensed from Santhera Pharmaceuticals to Catalyst Pharmaceuticals, Inc. for North America. In July 2024, Kye Pharmaceuticals entered into an agreement to sublicense from Catalyst the rights to commercialize AGAMREE^® in Canada.

"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."

News of the AGAMREE^® approval was welcomed by Canadian DMD patient organizations. 

"It's very impactful knowing that the first medicine approved in Canada is one that Defeat Duchenne Canada (formerly Jesse's Journey) supported through research." John Davidson, Founder of Jesse's Journey and current board member for Defeat Duchenne Canada.

"No one was prepared for our son Jesse's Duchenne diagnosis and our world turned upside down. There are several feelings that you experience and eventually you come to the realization that all of this bitterness and anger are not going to accomplish anything. So, we got to work," he continued.

Defeat Duchenne Canada is the only national organization focused solely on Duchenne Muscular Dystrophy in Canada. This year, in their 30th anniversary year, they will surpass investing 20 million dollars into research. Defeat Duchenne Canada helped to fund one of the open label studies for AGAMREE. This data in children aged 2-4 and age 7-17 years included boys from five different trial sites across Canada and at an age range often neglected in Duchenne clinical trials. This Canadian data supported the Health Canada approval and broad indication.⁶

"While corticosteroids have been a key treatment for decades, their significant side effects have been a challenge for the Duchenne muscular dystrophy community," says Stacey Lintern, Chief Executive Officer, Muscular Dystrophy Canada. "AGAMREE^® offers a treatment option that delivers efficacy while minimizing these side effects."

"Canada has played a critical role in this milestone, with clinicians, study teams, individuals, and families contributing to clinical trials that demonstrated the drug's safety and effectiveness. This approval highlights the power of collaboration and innovation in improving outcomes for those living with Duchenne and marks an exciting new chapter for Duchenne care in Canada," she continues.

About AGAMREE^® (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 AGAMREE^® 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, AGAMREE^® 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.⁵

About Kye Pharmaceuticals
Kye is a leading growth-stage 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 2025 Canada's Top Growing Companies list.

For more information about the company, its management, portfolio, pipeline and commitment, please visit  www.kyepharma.com

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SOURCE Kye Pharmaceuticals Inc.