Santhera Announces NDA for Vamorolone in Duchenne Muscular Dystrophy Accepted and Granted Priority Review by China’s NMPA

Pratteln, Switzerland, March 27, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces 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 Q1 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 dystrophy 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 in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment 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.

According to the license agreement between the companies, first announced in January 2022, Sperogenix Therapeutics holds exclusive development and commercialization rights to vamorolone in DMD and all other rare disease indications for China. Upon commercialization, Sperogenix will pay Santhera sales-based milestones and double-digit percentage royalties on net sales.

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].