Vamorolone up for approval in Canada as treatment for DMD
Health Canada grants priority review; therapy approved in US as Agamree
Health Canada has agreed to review an application by Kye Pharmaceuticals seeking the approval of vamorolone for treating Duchenne muscular dystrophy (DMD) — and granted it priority review.
Priority review shortens the review period from the standard 10 months to six. It’s given to therapies that have the potential to provide significant benefits in treating serious conditions. A final decision on vamorolone’s approval in Canada could be announced before the end of the year.
“Health Canada granting vamorolone a priority review is a promising first step for patients with Duchenne having access to approved medicines in Canada. If approved, vamorolone will provide Duchenne families access to an approved therapy that will positively impact their disease,” Jean Mah, MD, director of the pediatric neuromuscular program at Alberta Children’s Hospital in Calgary, said in a company press release.
This follows the therapy’s approvals in the U.S. and the U.K., and in the European Union, where it’s marketed under the brand name Agamree. If approved, vamorolone will become the first treatment available for people with DMD in Canada.
Lisa McCoy, CEO of Defeat Duchenne Canada, noted that, to date, no medications have been approved in the country for DMD patients.
“We urge Health Canada to approve Agamree to ensure all Canadians for whom this treatment would be beneficial can access it as soon as possible,” McCoy said.
Vamorolone also available under brand name Agamree in UK, EU
DMD is caused by mutations in the DMD gene that result in the lack of dystrophin, a protein that protects muscle cells from damage during movements. That lack of dystrophin results in progressive muscle weakness and wasting, usually starting in the muscles closer to the trunk.
As of now, in Canada, Duchenne is typically treated with corticosteroids, a type of anti-inflammatory medication. Used as standard of care to preserve muscle function and ease inflammation in people with DMD, such medications work by mimicking the effects of a hormone called cortisol. However, their long-term use can lead to a wide range of side effects, including impaired growth, weight gain, and fragile bones.
Vamorolone is an oral dissociative corticosteroid that interacts with cortisol receptors in a way that promotes anti-inflammatory pathways but avoids ones linked to the side effects seen with common corticosteroids.
In collaboration with our Canadian sub-licensee, Kye, [Catalyst Pharmaceuticals remains] committed to bringing this innovative therapy to DMD patients and ensuring access, particularly for those in underserved communities.
The therapy was originally developed by Reveragen Biopharma. In 2023, Catalyst Pharmaceuticals obtained vamorolone’s commercial rights in North America. Kye Pharmaceuticals has since acquired the rights to market the therapy in Canada.
“We are pleased that Health Canada has accepted the new drug submission for Agamree with a priority review, recognizing its potential to address a significant unmet need for patients with Duchenne muscular dystrophy in Canada,” Richard J. Daly, Catalyst’s president and CEO, said in a separate company press release. “In collaboration with our Canadian sub-licensee, Kye, we remain committed to bringing this innovative therapy to DMD patients and ensuring access, particularly for those in underserved communities.”
In other countries, approvals were based on data from the pivotal Phase 2b VISION-DMD trial (NCT03439670). That study’s results showed vamorolone was comparable to the standard corticosteroid prednisone in its ability to preserve muscle function, but with fewer side effects.
John McKendry, Kye’s president and CEO, called the submission “an important milestone for DMD treatments in Canada.”
“We look forward to working collaboratively with the DMD community and Health Canada throughout the priority review process,” McKendry said.
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