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Santhera receives positive recommendation from the Scottish Medicines Consortium for the use of AGAMREE® (Vamorolone) in patients with Duchenne muscular dystrophy in NHS Scotland

Santhera receives positive recommendation from the Scottish Medicines Consortium for the use of AGAMREE® (Vamorolone) in patients with Duchenne muscular dystrophy in NHS Scotland

  • AGAMREE ® is approved by the MHRA for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older in the United Kingdom

Pratteln, Switzerland, January 14, 2025 “ Santhera Pharmaceuticals (SIX: SANN) Announces that the Scottish Medical 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 aged 4 years and older.

Dario Eklund, CEO of Santhera, commented: “I am delighted that the team has secured this latest approval, which will ensure that Scottish patients can benefit from this important treatment for DMD. We will be working closely with NHS Scotland, along with all key regulatory and health authorities on all our approved markets, so that patients have access to AGAMREE.

AGAMREE was the first drug for DMD to be approved 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 new 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 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug enhancement and corticosteroid-related toxicity in local tissues [1-4]. This mechanism has shown the potential to “uncouple” efficacy from steroid safety concerns and therefore positions AGAMREE as a dissociative anti-inflammatory drug and alternative to existing corticosteroids, the current standard of care for children and adolescents with DMD [1-4].

In the pivotal VISION-DMD study, AGAMREE reached the primary endpoint Time to Stand (TTSTAND) rate versus placebo (p=0.002) after 24 weeks of treatment and demonstrated a good safety and tolerability profile [1,  4]. The most commonly reported side effects were cushingoid features, vomiting, weight gain and irritability. Adverse events were generally mild to moderate in severity.

Currently available data indicate that AGAMREE, unlike corticosteroids, does not have growth restriction [5] and no negative effects on bone metabolism as demonstrated by normal bone formation and serum markers for bone resorption [6].

–¼ This medicine is subject to additional monitoring. This will enable quick identification of new security information. Healthcare professionals are asked to report any suspected side effects.

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., Poster presentation MDA 2022. Link.

About Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a rare X-linked inherited disease that almost exclusively affects males. DMD is characterized by inflammation that is present at birth or shortly thereafter. Inflammation leads to muscle fibrosis, and is clinically manifested by progressive degeneration and muscle weakness. The main milestones in the disease are the loss of the ability to move, the loss of independent feeding, the start of assisted ventilation and the development of cardiomyopathy. DMD reduces life expectancy before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for DMD.

About Santhera
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 needs. The company holds an exclusive license from ReveraGen for all indications worldwide for AGAMREE ® (vamorolone), a dissociative steroid with a novel mode of action, which has been 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 US 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 (TASE:) Products Authority (NMPA) and Hong Kong by the Department of Health (DoH). Santhera has externally licensed rights to AGAMREE for North America at Catalyst Pharmaceuticals (NASDAQ:), and for China and certain countries in Southeast Asia Sperogenix Therapeutics. For further information, visit www.santhera.com.

AGAMREE ® is a trademark of Santhera Pharmaceuticals.

For additional information, please contact:
public-relations@santhera.com or
Elodie Denjean, Managing Director UK, France and Benelux
elodie.denjean@santhera.com or +44 7437865600.

Disclaimer / Forward-Looking Statements
This communication does not constitute an offer or invitation to subscribe or purchase securities Santhera Pharmaceuticals Holding (SIX:) AG. This publication may contain certain forward-looking statements regarding the company and its operations. Such statements involve certain risks, uncertainties and other factors that could cause the Company’s actual results, financial condition, performance or achievements to differ materially from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

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  • 250113 Issue of SMC approval_FINAL_ENGLISH

Source: Santhera Pharmaceuticals Holding AG





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