Health Canada has approved Kye Pharmaceuticals’ AGAMREE (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in boys age 4 and older.
The approval – granted under Health Canada’s Priority Review process – makes 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.
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. 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.
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, and fractures, among others, often prevent them from being able to tolerate the recommended dose of these medications.
“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 favourable changes in serum bone turnover biomarkers, it provides families with a viable treatment option compared to other corticosteroids,” said 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 behaviour.
AGAMREE is approved in the US, EU, UK, and China. It 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 commercialise 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,” said 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.”
“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,” said John Davidson, founder of Jesse’s Journey and board member for Defeat Duchenne Canada.
Defeat Duchenne Canada is the only national organisation focused solely on DMD in Canada. This year, in
“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, CEO, Muscular Dystrophy Canada.
“AGAMREE offers a treatment option that delivers efficacy while minimising 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.”
Jim Cornall is editor of Deeptech Digest and publisher at Ayr Coastal Media. He is an award-winning writer, editor, photographer, broadcaster, designer and author. Contact Jim here.
