Santhera Receives Positive Recommendation from Scottish Medicines Consortium for the Use of AGAMREE® (Vamorolone) in Duchenne Muscular Dystrophy Patients in NHS Scotland

• AGAMREE® is approved by MHRA for treating Duchenne muscular dystrophy (DMD) in patients 4 years of age and older in UK

Pratteln, Switzerland, January 14, 2025 – Santhera Pharmaceuticals (SIX: SANN) 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.

For more information about AGAMREE:   For Great Britain, including Scotland: Summary of Product Characteristics

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 [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].

▼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.

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.

About Duchenne Muscular Dystrophy 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 self-feeding, 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.

About SantheraSanthera 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 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.

AGAMREE® is a trademark of Santhera Pharmaceuticals.

For further information please contact: public-relations@santhera.com or Elodie Denjean, General Manager UK, France & Benelux elodie.denjean@santhera.com or +44 7437865600.

Disclaimer / Forward-looking statements This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

# # #

Attachment

• 250113 SMC approval release_FINAL_ENGLISH