Health Canada has approved Santhera Pharmaceuticals’ Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in individuals aged four years and above.

Agamree is the first approved therapy for DMD in the country.

The decision follows a priority review and is supported by the Phase IIb VISION-DMD trial data, along with safety data from four open-label trials, including extensions.

The studies evaluated the drug at doses between 2 to 6 mg/kg/day over periods up to 48 months.

Agamree's efficacy was on par with traditional corticosteroids, but with fewer adverse effects, particularly regarding bone health, growth and behaviour.

In July 2024, Kye Pharmaceuticals secured exclusive commercial rights for Agamree in Canada, following a sub-licence agreement with Santhera’s commercialisation partner, Catalyst Pharmaceuticals.

Kye Pharmaceuticals president and CEO John McKendry stated: “This Health Canada approval reflects Kye Pharmaceuticals’ commitment to advancing treatments for rare diseases and serving the unmet needs of Canadian patients.

“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.”

Santhera will obtain royalties from Canadian sales, which will also contribute to the sales milestones from Catalyst for the North American market.

The company has out-licensed the drug’s rights to Sperogenix Therapeutics for the Chinese market and certain Southeast Asian countries. 

During VISION-DMD, the drug met the primary goal by demonstrating an improvement in the time to stand velocity after 24 weeks of treatment when compared to a placebo, along with a favourable tolerability and safety profile. 

Santhera's focus is on developing and commercialising treatments for rare neuromuscular conditions that lack adequate medical solutions.

It holds an exclusive global licence from ReveraGen for all indications related to Agamree.

The US Food and Drug Administration, China’s National Medical Products Administration, Hong Kong’s Department of Health, the European Commission and the UK Medicines and Healthcare products Regulatory Agency also approved Agamree for DMD treatment.

Santhera previously signed an exclusive agreement with Ikris Pharma Network to distribute Agamree in India.