Health Canada approves Santhera’s Agamree, Canada’s first DMD treatment

CANADA—Health Canada has approved Santhera Pharmaceuticals’ drug Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients aged four years and older, making it the first approved treatment for DMD in Canada.

The approval follows a priority review based on data from the Phase IIb VISION-DMD trial and safety data from four additional open-label studies, including extensions.

These trials tested doses ranging from 2 to 6 mg/kg per day over periods of up to 48 months.

Agamree demonstrated effectiveness comparable to traditional corticosteroids but with fewer side effects, particularly concerning bone health, growth, and behavioral issues.

In July 2024, Kye Pharmaceuticals acquired exclusive commercial rights for Agamree in Canada through a sub-licensing deal with Catalyst Pharmaceuticals, Santhera’s commercialization partner.

John McKendry, President and CEO of Kye Pharmaceuticals, emphasized their dedication to improving rare disease treatments and addressing the unmet needs of Canadian patients.

He also highlighted their commitment to collaborating with the DMD community, provincial drug plans, and private insurers to ensure the drug’s accessibility.

Santhera will receive royalties from Canadian sales of Agamree, which will also contribute to sales milestones linked to its partnership with Catalyst in the North American market.

Additionally, Santhera has licensed Agamree’s rights for China and certain Southeast Asian countries to Sperogenix Therapeutics.

During the VISION-DMD trial, Agamree met its primary goal by demonstrating a significant improvement in standing velocity after 24 weeks compared to the placebo. The drug also displayed a favorable safety and tolerability profile.

Santhera focuses on developing therapies for rare neuromuscular diseases that currently lack effective treatments.

It holds an exclusive global license for Agamree from ReveraGen, covering all medical indications.

Agamree has also received approval from regulatory agencies, including the US Food and Drug Administration (FDA), China’s National Medical Products Administration, Hong Kong’s Department of Health, the European Commission, and the UK Medicines and Healthcare products Regulatory Agency.

Moreover, Santhera signed an exclusive distribution agreement with Ikris Pharma Network for Agamree in India.

The drug’s approval in multiple regions reflects growing recognition of its potential to improve the quality of life for people living with DMD worldwide.

Duchenne muscular dystrophy is a severe genetic disorder characterized by progressive muscle weakness, primarily affecting young boys.

Current corticosteroid treatments have been effective but often cause significant side effects, which Agamree aims to reduce.

Agamree’s development represents a significant step toward safer and more tolerable treatment options for patients with this condition.