Santhera Receives Positive Recommendation from Scottish Medicines Consortium for the Use of AGAMREE® (Vamorolone) in Duchenne Muscular Dystrophy Patients in NHS Scotland
January 14, 2025 – Other, Pharmaceutical – Duchenne Muscular Dystrophy, NHS Scotland, Santhera Pharmaceuticals, Scottish Medicines Consortium, neuromuscular diseases
AGAMREE® is approved by MHRA for treating Duchenne muscular dystrophy (DMD) in patients 4 years of age and older in UK
14 January 2025 — Pratteln, Switzerland — Santhera Pharmaceuticals announces that the Scottish Medicines Consortium (SMC) has published recommendations that AGAMREE® (vamorolone) is accepted for use within NHS Scotland for the treatment of Duchenne muscular dystrophy (DMD) in patients 4 years of age and older.
Dario Eklund, CEO of Santhera, commented: “I am delighted that the team has secured this latest approval, which will ensure Scottish patients can benefit from this important treatment for DMD. We will work closely with NHS Scotland, along with all the key regulatory and health authorities in all our approved markets, to ensure patients have access to AGAMREE.”
AGAMREE was the first medicinal product for DMD to have received approval in the EU, US, and UK.
About AGAMREE® (vamorolone)
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. 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.
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. 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 and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers.
About Santhera
Santhera Pharmaceuticals 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 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 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. For further information, please visit www.santhera.com.
