On December 11, 2024, Shufang Medicine announced the announcement of Anjiali ® (Common name: Vamorolone Oral Suspension, Vamorolone); Specification: 40mg/mL, 100mL/bottle) has recently been approved by the National Medical Products Administration (NMPA) of China for the treatment of Duchenne muscular dystrophy (DMD) patients aged four years and above. Angali ® It is the first domestically approved treatment drug for Duchenne muscular dystrophy. Angali ® Previously, it was included in the breakthrough treatment drug and priority review procedures by the Drug Evaluation Center of the National Medical Products Administration.Duchenne muscular dystrophy is a rare neuromuscular disease that often occurs in childhood, and there is a huge unmet medical need in this field. Angali ® It is the first DMD treatment drug that has been fully approved in both the United States and the European Union. Its receptor that binds to glucocorticoids is the same, but its downstream activity is altered. Compared with traditional corticosteroid treatment, it not only has comparable efficacy but also has important clinical safety advantages in maintaining normal bone metabolism, bone density, and growth. Therefore, Angali ® Expected to become the standard treatment for DMD patients.

Mr. Yan Zhiyu, co-founder, chairman, and CEO of Shufang Pharmaceutical, said, "Anjiali ® The rapid approval through the priority review process reflects the high importance that the country attaches to the research and development of rare disease drugs, as well as the firm commitment of Shufang to the group of rare disease patients who are eagerly awaiting treatment in China. Angali ® The successful approval of Shufang Pharmaceutical is an important milestone in its development. Moving forward, we will continue to prioritize patient needs and collaborate with relevant parties to accelerate supply and access efforts, allowing more families of DMD patients to benefit as soon as possible

Dr. Ang Qiuqing, Chief Medical and Development Officer of Shufang Pharmaceutical, stated: "For a long time, corticosteroid drugs that should have played a fundamental role in DMD treatment have been insufficiently treated due to their safety issues, resulting in a dual loss of clinical efficacy and quality of life ® The approval is expected to change this situation, giving DMD patients the opportunity to receive sufficient and long-term standardized treatment, improving their long-term prognosis and quality of life

About Anjiali ®

Angali ® It is an original new drug, which is a new type of dissociative corticosteroid. At the 9:11 position in the chemical structure, the hydroxyl group (OH) is changed to a carbon carbon double bond, making Angelic available ® While possessing strong anti-inflammatory properties, it also has better safety features.

In the critical study of Vision DMD, Angeli ® The efficacy of 6 mg/kg/day and sufficient prednisone 0.75 mg/kg/day is similar in all motor functions. The improvement persisted throughout the 48 week treatment period. 30 month long data from VBP15-LTE research shows that Angali ® The average TTSTAND speed from lying down to standing was consistently higher than the baseline value.

In terms of safety, sufficient amount of agaric ® Compared to sufficient traditional hormones, Angeli ® The frequency and severity of clinically related adverse reactions are low, and the incidence of adverse events such as behavioral abnormalities, diabetes, skin and hair changes, and infections is lower. There are no cataract events, and there are no harmful effects on bone metabolism and growth. Angali ® Better safety makes it possible to provide long-term and adequate standardized treatment for DMD.

Angali ® Currently approved in China, the United States, the European Union, and the United Kingdom.

Regarding Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a rare X-chromosome recessive inherited myopathy that primarily affects males. The pathogenic variation of DMD gene causes abnormal expression of anti muscle atrophy protein, leading to muscular dystrophy and inflammation, which usually occurs at birth or shortly after birth. Inflammation leads to muscle fibrosis, clinically manifested as symmetrical, progressive muscle degeneration and weakness. The main turning points of deterioration of this disease are loss of walking ability, loss of self feeding ability, initiation of assisted ventilation, and concurrent cardiomyopathy. Most patients die before the age of 30 due to respiratory or heart failure. Oral corticosteroids are currently the standard treatment for DMD, and long-term use can prolong patients' independent walking time by 2-5 years or more, prolong their lives, and improve cardiovascular function. However, research has shown that due to the numerous adverse reactions of glucocorticoids, the discontinuation rate of DMD patients in China is 39.6%; Only 54.1% of patients have been using a daily hormone regimen (prednisone/prednisolone 0.3-0.75 mg/kg/day) for more than 1 year. However, insufficient and non-standard use of hormones has been proven to result in a loss of therapeutic efficacy and inability to achieve optimal results.

About Shufang Medicine

Shufang Pharmaceutical was founded in 2019 and is an innovative enterprise dedicated to the research and commercialization of drugs for genetic diseases and rare diseases. It is committed to building an innovative business model that fits the characteristics of the Chinese rare disease market, and providing reliable products and services with clinical accessibility for doctors and patients. In 2022, Shufang Pharmaceutical obtained exclusive development and commercialization rights of famolon for indications of Duchenne muscular dystrophy and other rare diseases in China (including Hong Kong, Macau, and Taiwan) and Southeast Asia from Santhera Pharmaceuticals in Switzerland, as well as production rights under certain conditions in all of the aforementioned regions.

Shufang Pharmaceutical website: www.sperogenix.com

About Santhera Pharmaceuticals

Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company that focuses on the development and commercialization of innovative drugs for rare neuromuscular diseases, a field with highly unmet medical needs. The company has exclusive licenses for all indications of famolon worldwide, and has been approved by the US FDA, EU EMA, UK MHRA, and China NMPA for the treatment of DMD patients. Santhera has granted the North American rights of famolon to Catalyst Pharmaceuticals, and the rights in Greater China and Southeast Asia to Shufang Pharmaceuticals.

Santhera Pharmaceutical website: www.santhera.com