Vamorolone

Health Canada approved AGAMREE for the treatment of Duchenne muscular dystrophy in patients aged 4 years and older This approval makes AGAMREE the first and only approved therapy for DMD in Canada October 3, 2025 – Santhera Pharmaceuticals (SIX: SANN) today notes that Health Canada has approved AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older following Priority Review, marking the first approved treatment for the disease in Canada. 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. Kye Pharmaceuticals holds exclusive Canadian commercial rights to AGAMREE for the treatment of DMD and potentially other indications, following the agreement of a sub-license with Santhera’s commercialization partner Catalyst Pharmaceuticals, Inc. (“Catalyst”) in July 2024. Santhera will receive royalties on Canadian sales and these sales will also contribute towards North American sales milestones payable to Santhera from its partner Catalyst. This is the sixth independent approval a by local health authority, following positive regulatory decisions in the U.S., Europe, the UK, China and Hong Kong. Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of selffeeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. AGAMREE is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Moreover, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues [1-4]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore AGAMREE is positioned as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [1-4]. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [1, 4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that AGAMREE, unlike corticosteroids, has no restriction of growth [5] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [6]. References: [1] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link. [2] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [3] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293 [4] Heier CR et al (2019). Life Science Alliance DOI: 10.26508 [5] Ward et al., WMS 2022, FP.27 - Poster 71. Link. [6] Hasham et al., MDA 2022 Poster presentation. Link. Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Commission (EC), in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA), in China by the National Medical Products Administration (NMPA) and Hong Kong by the Department of Health (DoH). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China and certain countries in Southeast Asia to Sperogenix Therapeutics. Kye is a leading growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of innovative medicines, Kye’s portfolio spans a range of therapeutic areas including cardiology, psychiatry, pediatrics, rare diseases, neuromuscular, hematology, and neurology. Kye Pharmaceuticals is a private company, founded in Canada and focused exclusively on the healthcare needs of patients in Canada. Kye aims to provide medicines that deliver better outcomes to our partners, Canadian healthcare professionals, and most importantly, patients across Canada. Kye, headquartered in Mississauga, Ontario was recognized on the Globe & Mail 2025 Canada’s Top Growing Companies list. The content above comes from the network. if any infringement, please contact us to modify.

Agamree has secured approvals from several countries for Duchenne muscular dystrophy treatment. Credit: MY STOCKERS/Shutterstock.com. 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. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence 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. Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now