#Canada #Mississauga #Duchenne #AGAMREE #Kye Pharmaceuticals

Health Canada Grants Approval for AGAMREE®

Kye Pharmaceuticals recently announced that Health Canada has approved AGAMREE® (vamorolone) for the treatment of Duchenne Muscular Dystrophy (DMD) in boys aged four and older. This momentous decision, reached under the Priority Review process, signifies the introduction of the first therapeutic option for DMD patients in Canada—a condition known for its severe impact on young boys.

DMD is a rare and debilitating neuromuscular disorder that deprives muscle cells of a crucial protein called dystrophin, leading to muscle weakness and wasting. The absence of dystrophin means that affected individuals face significant challenges in essential movements, such as standing up and walking. Typically, boys with DMD lose their ability to walk by their early teenage years, and they also face increased risk of cardiac and respiratory complications. As per the Canadian Neuromuscular Disease Registry, over 800 boys and young men in Canada are grappling with the profound effects of DMD.

The standard treatment for DMD has been corticosteroids, which can prolong the ability to walk for a few more years. However, these medications often come with unpleasant side effects, including weight gain, growth stunting, and behavioral issues that make the treatment difficult to tolerate for some patients.

Dr. Jean Mah, who oversees the Pediatric Neuromuscular Program at the Alberta Children's Hospital in Calgary, expressed his satisfaction with the approval, citing it as a key step for those with DMD. He pointed out that AGAMREE® does not negatively impact linear growth and shows positive changes in indicators of bone health, offering families a viable treatment alternative compared to traditional corticosteroids.

The endorsement of AGAMREE® is a result of findings from the pivotal Phase 2b VISION-DMD study alongside data gathered from several open-label studies. These trials subjected boys to AGAMREE® dosages between 2 to 6 mg/kg/day for as long as 48 months. The outcome demonstrated comparable efficacy to standard care corticosteroids while minimizing adverse effects, particularly those impacting bone health and growth.

Alongside its Canadian approval, AGAMREE® is already recognized in markets such as the US, EU, UK, and China. Originally developed by Santhera Pharmaceuticals, the product rights were licensed to Catalyst Pharmaceuticals for the North American region. Kye Pharmaceuticals secured the commercialization rights for Canada in July 2024.

John McKendry, President and CEO of Kye Pharmaceuticals, spoke about the company's dedication to addressing rare disease treatment needs in Canada. He emphasized the aim to work in close coordination with the DMD community and existing health systems to ensure access to AGAMREE®.

This significant milestone was welcomed by Canadian organizations advocating for DMD patients. John Davidson, founder of Defeat Duchenne Canada, highlighted that the organization has been actively involved in supporting research related to AGAMREE®. Returning to personal experiences, he recounted the emotional turmoil following his son's DMD diagnosis and how that impelled him to action.

Kye Pharmaceuticals is proud to have played a role in this progression, with Defeat Duchenne Canada stating that they have invested over 20 million dollars in DMD research, with part of these funds being allocated toward studies for AGAMREE®.

Stacey Lintern, CEO of Muscular Dystrophy Canada, noted the profound differences that AGAMREE® could make in the lives of DMD patients, given the historical side effects of existing corticosteroids.

The structure of AGAMREE® is unique, functioning through differential effects on glucocorticoid and mineralocorticoid receptors, tailoring its activity. This novel mechanism could allow it to emerge as a strong alternative to traditional steroids. In the VISION-DMD study, AGAMREE® was notably effective, showing improved functional outcomes in clinical trials while maintaining a favorable safety profile. Reports of mild to moderate adverse effects were relatively few compared to those seen with other treatments.

Kye Pharmaceuticals has established itself as a prominent Canadian specialty pharmaceutical company, aspiring to meet patients' healthcare needs through innovative medicines. The company is continuing its dedication to enhancing the quality of life and health outcomes for individuals living with DMD and other rare diseases.

For additional information regarding Kye Pharmaceuticals and its endeavors, please visit Kye Pharmaceuticals.