December 11, 2024, Sperogenix Therapeutics announced that, AGAMREE® (vamorolone) was approved by China’s National Medical Products Administration (NMPA) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older, which is the first and only approved DMD therapy in China. AGAMREE® has been included in the “Breakthrough Therapy Program” and “Priority Review Program” by the Center for Drug Evaluation (CDE) of China.
DMD is a rare inherited X-chromosome-linked neuromuscular disease, often has its onset in childhood, and there is a huge unmet medical need in this field. As the first fully approved treatment for DMD in both the United States (2 years and older) and the European Union (4 years and older), AGAMREE® is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Compared with traditional corticosteroids, AGAMREE® not only has equal efficacy but also has important clinical safety advantages in maintaining normal bone metabolism, bone mineral density and growth. Therefore, AGAMREE® could emerge as an alternative to existing corticosteroids, the current standard of care in patients with DMD.
Mr. Yan Zhiyu, Co-founder, Chairman and CEO of Sperogenix Therapeutics, said, “The approval of AGAMREE® through the Priority Review Program reflects the government's high level of attention to the development of rare disease drugs, and also reflects Sperogenix's firm commitment to the rare disease patients in China who are in urgent unmet needs. The approval of AGAMREE® is an important milestone, and we will continue to uphold our commitment to patient needs and work with stakeholders to accelerate supply and access initiatives, so that more DMD patient families can benefit as soon as possible.”
Dr. Ang Qiuqing, Chief Medical and Development Officer of Sperogenix Therapeutics, said, “For a long time, corticosteroid drugs that should play a fundamental role in the treatment of DMD have led to inadequate treatment due to their safety issues, resulting in a double loss of clinical effect and quality of life. The approval of AGAMREE® is expected to change this, providing DMD patients with the opportunity to receive long-term, adequate and standardized treatment to improve their long-term prognosis and quality of life.”
About AGAMREE® (Vamorolone) [EU1]
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].
AGAMREE® (vamorolone), an orphan medicinal product, is approved for use in the People’s Republic of China (PRC), the United States (U.S.) (Prescribing Information), the European Union (EU) (Summary of Product Characteristics) and the United Kingdom (UK).
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 Sperogenix Therapeutics
Founded in 2019, Sperogenix Therapeutics is a platform company dedicated to developing and commercializing genetic disease and rare disease therapeutics in China. With prioritized therapeutic areas such as neuromuscular diseases and inherited metabolic diseases, Sperogenix is dedicated to establishing an innovative commercial model tailored to the China rare disease field, in order to provide affordable and reliable products and services to Chinese physicians and patients. In 2022, Sperogenix Therapeutics obtained the exclusive business development and commercialization rights from Santhera Pharmaceuticals for AGAMREE® in DMD and all other rare diseases in the Greater China region (including Hong Kong, Macao and Taiwan) and the Southeast Asian region and production rights in all of the above regions under certain conditions. www.sperogenix.com.
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 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), and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics. For further information, please visit www.santhera.com.
AGAMREE® is a trademark of Santhera Pharmaceuticals.
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.
Source: Sperogenix