On September 16, 2023, the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China announced that the new drug vamorolone sponsored by Sperogenix Therapeutics and Santhera Pharmaceuticals was included in the "breakthrough therapy" program, and the proposed indication is for Duchenne muscular dystrophy (DMD) patients who are 2 years old and above.

According to the Announcement of China NMPA on the release of the "Breakthrough Therapy Review Procedure" and other documents [No. 82, 2020], the "breakthrough therapy" procedure refers to the prevention and treatment of diseases that seriously threaten life or seriously affect the quality of life. And there is no effective medications of prevention and treatment or there is sufficient evidence to show that there are obvious clinical advantages compared with existing treatment of innovative drugs or improved new drugs, the sponsors can apply for the application of breakthrough therapy program. The inclusion of drugs in the "breakthrough therapy" review channel means that CDE will prioritize the allocation of review resources for them, strengthen communication and guidance, and promote the development of new drugs.

Duchenne muscular dystrophy is a rare neuromuscular disease that affects about 1 in 4,560 male newborns in China and is estimated to affect about 100,000 people. There is currently no officially approved drug for DMD treatment in China, and there is a huge unmet medical need in this field. Positive clinical data from the pivotal clinical trial of Vamorolone (VISION-DMD) demonstrated that the trial met the primary endpoint Time to Stand (TTSTAND) velocity and other secondary endpoints, with the experimental group outperforming the placebo group with statistically significant differences. While vamorolone demonstrated a good safety and tolerability profile.

Previously, the vamorolone clinical trial application submitted by Sperogenix has been approval by CDE. In addition, vamorolone has submitted new drug marketing authorization applications in the United States and the European Union, respectively, and is expected to be approved in the United States in October 2023. Vamorolone has been granted Orphan Drug status for DMD in the U.S. and in Europe and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD.

On January 4, 2022, Sperogenix Therapeutics and Santhera Pharmaceuticals announced an exclusive licensing agreement for vamorolone in DMD and all other rare diseases in the Greater China region.

About Vamorolone

Vamorolone is an investigational drug candidate with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity and as such is considered a dissociative anti-inflammatory drug [2-5]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore vamorolone could emerge as an alternative to existing corticosteroids, the current standard of care in children and adolescent subjects with DMD. In the pivotal VISION-DMD study, vamorolone 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]. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity. Vamorolone is an investigational medicine and is currently not approved for use by any health authority.

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.

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 pulmonary vascular disorders, 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 Pharmaceuticals

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 need. The Company has an exclusive license for all indications worldwide to 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. For vamorolone in the treatment of DMD, Santhera has a new drug application (NDA) under review by the U.S. FDA, a marketing authorization application (MAA) under review by the European Medicines Agency (EMA) and an MAA submitted to the UK Medicines and Healthcare products Regulatory Agency (MHRA). The clinical stage pipeline also includes lonodelestat to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases. Santhera out-licensed rights to its first approved product, Raxone® (idebenone), outside North America and France for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group. For further information, please visit www.santhera.com.

References:

[1] Guglieri M et al. (2022). JAMA Neurol. Published online August 29, 2022. The doi: 10.1001 / jamaneurol. 2022.2480.

[2] Mah JK et al (2022). JAMA Netw Open. 2022; 5 (1) : e2144178. Doi: 10.1001 / jamanetworkopen. 2021.44178.

[3] Guglieri, et al. (2022), JAMA, doi: 10.1001 / JAMA. 2022.4315

[4] Heier CR at al(2019). Life Science Alliance DOI: 10.26508

[5] Liu X, et al (2020). Proc Natl Acad Sci USA 117:24285-24293

[6] https://www.nmpa.gov.cn/yaopin/ypggtg/ypqtgg/20200708151701834.html

[7]https://www.cde.org.cn/main/news/viewInfoCommon/3d0db20b8d9aa0e73808e60b9529b97c