March 26, 2024, the NDA filling of AGAMREE (vamorolone) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older has been accepted by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA), and been included in the “Priority Review Program. In 2023, based on its safety, efficacy, and significant clinical advantages over existing treatments, AGAMREE was included in the "Breakthrough Therapy Program " by CDE.

DMD is a rare inherited X-chromosome-linked neuromuscular disease, there is currently no officially approved drug to treat DMD in China, and there is a huge unmet medical need in this field. As the first fully approved treatment for DMD in both the United States and the European Union, 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.

Dr. Ang Qiuqing, Chief Medical Officer of Sperogenix Therapeutics, said, “NDA is an important milestone in the development of AGAMREE. Thanks to the incentive policy for innovative drugs for rare diseases in China, we will actively cooperate with regulatory authorities to accelerate the registration, development and clinical application to benefit Chinese DMD patients as soon as possible.”

Mr. Wang Xiaohui, Co-founder and Chief Business and Operations Officer of Sperogenix Therapeutics, said, “AGAMREE is a beneficial try by Sperogenix to explore a rapid commercialization innovation path for rare disease drugs based on the characteristics of the Chinese market. While speeding up the registration, we are in close communication with medical institutions in the Boao Lecheng International Medical Tourism Pilot Zone in Hainan province, accelerating the accessibility of AGAMREE in Lecheng to respond to the urgent treatment needs of patients.”

In 2022, Sperogenix Therapeutics obtained the exclusive business development and commercialization rights from Swiss Santhera Pharmaceuticals for AGAMREE in DMD and all other rare diseases in the Greater China region (including Hong Kong, Macao and Taiwan), and some business development and commercialization rights in the Southeast Asian region and production rights in all of the above regions under certain conditions.

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 of statistical significance in 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 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. Pathogenic variants of the DMD gene cause abnormal expression of dystrophin, leading to the development of muscular dystrophy and 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.

About Sperogenix Therapeutics

Sperogenix Therapeutics is a platform company dedicated to developing and commercializing 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. Sperogenix was founded in 2019 and is backed by biopharma industry blue chip investors including Lilly Asia Ventures, Morningside Ventures and Prosperico Ventures. 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 and pulmonary diseases with high unmet medical needs. 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 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, Inc. and for China to Sperogenix Therapeutics. The clinical stage pipeline also includes lonodelestat to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases. 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.