Pratteln, Switzerland, April 16, 2025 Santhera Pharmaceuticals (SIX: SANN) has announced significant advancements in the commercial rollout of AGAMREE, a groundbreaking treatment for Duchenne Muscular Dystrophy (DMD). This announcement follows a series of encouraging developments aimed at improving the lives of patients suffering from this severe genetic disorder.

One of the most notable updates is the availability of funding from National Health Service (NHS) England, which will now facilitate the prescription of AGAMREE to patients in England. This pivotal step comes on the heels of a positive ruling from the National Institute for Health and Care Excellence (NICE), which provided final guidance for AGAMREE on January 16, 2025. This approval marks a significant milestone, as initial sales of the medication have already taken place in Scotland and Wales, indicating a growing acceptance of the drug within the UK healthcare system.

Additionally, Santhera has shared that Kye Pharmaceuticals, a sub-licensee of its commercialization partner Catalyst Pharmaceuticals, has received Health Canadas acceptance for the New Drug Submission concerning AGAMREE, which has been granted Priority Review status. This acceptance is particularly noteworthy as it potentially leads to AGAMREE becoming the first approved treatment for DMD in Canada by the end of 2025, providing hope for Canadian patients and their families who have long awaited effective therapeutic options.

Dario Eklund, the CEO of Santhera, expressed his satisfaction with the latest developments, stating: "Im pleased to share the latest progress on the commercial rollout of AGAMREE, particularly in the UK, where a significant portion of our clinical trials were conducted. These trial sites played a key role in generating the data to support the approval. We are proud to see this milestone translate into access for patients in England living with DMD." His remarks signify not only the companys commitment to patient care but also highlight the collaborative efforts that led to this advancement.

AGAMREE (vamorolone) is an innovative therapeutic option designed to address the needs of patients with DMD. Its unique mode of action involves binding to the same receptor as glucocorticoids, while simultaneously modifying its downstream activities to enhance therapeutic efficacy and minimize adverse effects. Importantly, AGAMREE is not affected by the 11--hydroxysteroid dehydrogenase (11-HSD) enzymes, which are often involved in local drug amplification and associated corticosteroid toxicity. This distinctive mechanism positions AGAMREE as a dissociative anti-inflammatory agent, making it an attractive alternative to traditional corticosteroids that have been the standard treatment for children and adolescents suffering from DMD.

In clinical trials, such as the pivotal VISION-DMD study, AGAMREE demonstrated promising results by meeting the primary endpoint of Time to Stand (TTSTAND) velocity versus placebo (p=0.002) after 24 weeks of treatment. The drug has also shown a favorable safety and tolerability profile, with commonly reported side effects including minor cushingoid features, vomiting, weight gain, and irritability. These side effects were generally mild to moderate, a notable contrast to the often severe side effects associated with traditional corticosteroid treatments.

Current evidence suggests that AGAMREE does not restrict growtha critical consideration for young patientsand has no detrimental effects on bone metabolism, as indicated by normal serum markers for bone formation and resorption. This characteristic is particularly important for children with DMD, who are at a higher risk of developing bone-related complications.

It is essential to note that AGAMREE is subject to additional monitoring, which allows for the swift identification of new safety information. Healthcare professionals are encouraged to report any suspected adverse reactions to ensure the ongoing safety and efficacy of the product.

Santhera Pharmaceuticals, headquartered in Switzerland, is dedicated to developing and commercializing innovative treatments for rare neuromuscular diseases, addressing significant unmet medical needs. The company holds an exclusive license from ReveraGen for AGAMREE (vamorolone) for all indications worldwide. AGAMREE has received regulatory approvals in various regions, including the U.S. by the Food and Drug Administration (FDA), the EU by the European Medicines Agency (EMA), and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). Furthermore, rights to AGAMREE have been out-licensed to Catalyst Pharmaceuticals for North America and to Sperogenix Therapeutics for China and select Southeast Asian countries.

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