Santhera Pharmaceuticals announced that the China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) which was submitted by Sperogenix Therapeutics, Santhera's specialized rare disease partner for China. The Center for Drug Evaluation (CDE) of the Chinese drug authority NMPA accepted the filing and granted priority review for vamorolone In DMD for patients aged 4 years and older which could, subject to a positive outcome, lead to approval by First Quarter 2025. Previously, the CDE included vamorolone for the treatment of DMD in the Breakthrough Therapy Program, which addresses serious diseases lacking effective treatments and includes drugs offering clear clinical advantages over existing treatments.

Duchenne muscular dystro Murphy is a rare neuromuscular disease affecting about 70,000 patients in China. Currently, there is no approved drug to treat DMD in China, leaving a high unmet medical need and therapeutic gap, especially considering the increasing diagnosis rates that enable more patients to access specialized treatment centers. The NDA in China is supported by an extensive data package which led to approval of vamorolone in the U.S., EU and UK in late 2023/early 2024.

Evidence for clinical efficacy and safety was based on data from the positive pivotal VISION-DMD study, showing superiority of vamorolone compared with placebo, and three open-label studies in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months. In addition, the filing included data from three open-label studies inwhich vamorolone wasadministered at doses between 2 and 6 ukg/day for a totaltreatment period of up to 30 months [1-4]. The submission is further supported by a study which investigated the pharmacokinetic parameters of vamorolone In healthy adult Chinese volunteers.

Duchenne muscular dy Strophy (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.orticosteroids are the current standard of care for the treatment of DMD.