Santhera Pharmaceuticals Makes Significant Progress with AGAMREE for Duchenne Muscular Dystrophy Treatment
Pratteln, Switzerland, 16 April 2025 - Santhera Pharmaceuticals (SIX: SANN) has announced a significant advancement in the commercial rollout of AGAMREE, a treatment for Duchenne Muscular Dystrophy (DMD). This announcement comes as the National Health Service (NHS) in England has now allocated financial resources for the prescription of AGAMREE, paving the way for its availability to patients in England. This development follows a positive final recommendation from the National Institute for Health and Care Excellence (NICE) for AGAMREE, which was publicly disclosed on January 16, 2025. Notably, initial sales of the drug have already been achieved in Scotland and Wales.
In addition to the developments in the UK, Santhera also reported that Kye Pharmaceuticals, a sublicensee of its marketing partner Catalyst Pharmaceuticals, has received approval from Health Canada to submit an application for AGAMREE under an expedited review process. This pivotal approval could potentially lead to AGAMREE becoming the first authorized treatment for DMD in Canada by the end of 2025.
Dario Eklund, CEO of Santhera Pharmaceuticals, expressed his enthusiasm regarding the recent progress made with AGAMREE, particularly in the UK where a significant portion of the clinical trials were conducted. He stated, I am pleased to announce the latest advancements in the launch of AGAMREE, especially in the United Kingdom, where several clinical trial centers played a crucial role in generating the data necessary for approval. We are proud that this milestone facilitates access for patients in England living with DMD.
AGAMREE (Vamorolone) represents a novel pharmaceutical approach with a unique mechanism of action that binds to the same receptor as glucocorticoids but modifies its downstream activity. This characteristic distinguishes it from typical corticosteroids, as AGAMREE is not a substrate for the 11--Hydroxysteroid-Dehydrogenase (11-HSD) enzymes, which are believed to be responsible for localized increased drug levels and corticosteroid-associated toxicity within tissues. This mechanism has the potential to uncouple efficacy from the safety concerns typically associated with steroid therapies, positioning AGAMREE as a dissociative anti-inflammatory agent and an alternative to existing corticosteroids, which are currently the standard treatment for children and adolescents with DMD.
In the pivotal VISION-DMD study, AGAMREE achieved its primary endpoint of time to stand (TTSTAND) when compared to a placebo (p=0.002) after 24 weeks of treatment, demonstrating a favorable safety and tolerability profile. The most commonly reported side effects included Cushingoid appearance, vomiting, weight gain, and irritability. Overall, the adverse effects were classified as mild to moderate in severity.
Data currently available indicate that, unlike corticosteroids, AGAMREE does not impair growth and shows no adverse effects on bone metabolism, as evidenced by normal serum markers for bone formation and resorption.
It is important to note that this medication is subject to additional monitoring, allowing for swift identification of new safety information. Healthcare professionals are encouraged to report any suspected side effects.
References:
- Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.
- Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link.
- Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293
- Heier CR et al (2019). Life Science Alliance DOI: 10.26508
- Ward et al., WMS 2022, FP.27 - Poster 71. Link.
- Hasham et al., MDA 2022 Poster presentation. Link.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a rare, hereditary, X-linked condition that primarily affects males. The disease is characterized by inflammation that occurs at birth or shortly thereafter, leading to muscle fibrosis and clinically manifesting as progressive muscle degeneration and weakness. Key milestones in the disease include the loss of ambulation, loss of self-feeding abilities, the initiation of assisted ventilation, and the development of cardiomyopathy. DMD significantly reduces life expectancy, often resulting in death before the third or fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard treatment for DMD.
About Santhera Pharmaceuticals
Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focusing on the development and commercialization of innovative drug therapies for rare neuromuscular diseases with high unmet medical needs. The company holds an exclusive worldwide license from ReveraGen for all indications of AGAMREE (Vamorolone), a dissociative steroid with a novel mechanism of action that has been investigated as an alternative to standard corticosteroids in a pivotal study involving patients with Duchenne Muscular Dystrophy (DMD). AGAMREE is approved for DMD treatment in the USA by the Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA), in China by the National Medical Products Administration (NMPA), and in Hong Kong by the Department of Health (DoH). Santhera has licensed the rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China and certain Southeast Asian countries to Sperogenix Therapeutics. For more information, visit
Mei-Ling Chen
