Santhera Pharmaceuticals announces that the health authorities of Hong Kong, China, have approved AGAMREE® (vamorolone) for the treatment of patients aged 4 years and older.

Following the US FDA, EMEA, MHRA and NMPA, the approval by the Hong Kong DoH is the fifth independent approval by local health authorities and will enable patients with DMD in Hong Kong to access AGAMREE in the near future.

According to the license agreement between the companies, which was first announced in January 2022 , Sperogenix holds exclusive development and commercialization rights for AGAMREE in DMD and other rare diseases in Greater China. This agreement was amended in March 2023 to include an option for several additional Southeast Asian countries, namely Brunei, Cambodia, Timor-Leste (East Timor), Indonesia, Laos, Malaysia, Myanmar (Burma), the Philippines, Singapore, Thailand and Vietnam, for a payment of USD 4 million by Sperogenix. Sperogenix exercised this option in July 2024 to include these territories in the license agreement. Santhera will supply AGAMREE to Sperogenix for both the ongoing Early Access Program (EAP) and commercialization. Sperogenix will pay Santhera double-digit royalties on net product sales as well as additional sales-based milestones based on commercial sales.

About AGAMREE® (vamorolone)

AGAMREE is a novel drug with a mechanism of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Furthermore, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes, which are thought to be responsible for locally increased drug levels and corticosteroid-associated toxicity in local tissues [1-4]. This mechanism has the potential to decouple efficacy from safety concerns associated with steroids, which is why AGAMREE is positioned as a dissociative anti-inflammatory 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 met the primary endpoint of standing-to-stand speed (TTSTAND) compared to placebo (p=0.002) after 24 weeks of treatment and demonstrated a favorable safety and tolerability profile [1, 4]. The most commonly reported adverse reactions were Cushingoid appearance, vomiting, weight gain and irritability. In general, adverse reactions were mild to moderate in severity.

Currently available data show that, unlike corticosteroids, AGAMREE does not restrict growth [5] and has no negative effects on bone metabolism, as demonstrated by normal serum markers of bone formation and resorption [6].

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 Posterpräsentation. Link.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a rare inherited disease linked to the X chromosome that affects almost exclusively males. DMD is characterized by inflammation that occurs at birth or shortly thereafter. The inflammation leads to fibrosis of the muscles and manifests clinically as progressive muscle degeneration and weakness. Major milestones of the disease include loss of walking, loss of self-feeding, initiation of assisted ventilation, and development of cardiomyopathy. DMD reduces life expectancy due to respiratory and/or cardiac failure before the fourth decade of life. 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 drugs for rare neuromuscular diseases with high unmet medical need. The Company holds an exclusive license from ReveraGen for all indications worldwide for AGAMREE® (vamorolone), a dissociative steroid with a novel mode of action that has been evaluated in a registration study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the United States by the U.S. Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), in the United Kingdom by the Medicines and Healthcare products Regulatory Agency (MHRA) and in China by the National Medical Products Administration (NMPA). Santhera has out-licensed the rights to AGAMREE to Catalyst Pharmaceuticals, Inc. for North America and to Sperogenix Therapeutics for China. For further information please visit www.santhera.de .

AGAMREE® is a trademark of Santhera Pharmaceuticals.

For further information please contact:
public-relations@santhera.com or
Andrew Smith, Chief Financial Officer
andrew.smith@santhera.com

About Sperogenix Therapeutics

Sperogenix Therapeutics is a platform company founded in 2019 and focused on the development and commercialization of therapeutics for genetic and rare diseases in China. Founded in 2019, Sperogenix is ​​a platform company dedicated to the development and commercialization of therapeutics for genetic and rare diseases in China. With priority therapeutic areas such as neuromuscular diseases and inherited metabolic diseases, Sperogenix is ​​dedicated to building an innovative business model tailored to the rare disease field in China to provide Chinese physicians and patients with affordable and reliable products and services. In 2022, Sperogenix obtained exclusive development and commercialization rights for AGAMREE® for DMD and all other rare diseases in the Greater China region (including Hong Kong, Macao and Taiwan) and the Southeast Asia region from Santhera Pharmaceuticals, as well as manufacturing rights in all of the above regions under certain conditions. www.sperogenix.com .

Disclaimer / Forward-looking statements

This communication does not constitute an offer or a solicitation to subscribe for or purchase securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements about the Company and its business activities. Such statements involve certain risks, uncertainties and other factors that may cause the actual results, financial position, performance or achievements of the Company 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 contracts or investment decisions. The Company disclaims any obligation to update these forward-looking statements.

 

Source: globenewswire.com

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