Sperogenix Therapeutics, a partner of the rare disease company in China, has applied with the National Medical Products Administration (NMPA) for Duchenne Muscular Dystrophy (DMD). The Center for Drug Evaluation (CDE) of the NMPA accepted the filing, having previously included vamorolone in their breakthrough therapy program. An approval decision for the use of vamorolone in treating patients aged four years and above with Duchenne Muscular Dystrophy is expected in the first quarter of next year.
The New Drug Application (NDA) in China is supported by a comprehensive data package that has already led to vamorolone’s approval in the US, EU, and UK. This data includes results from the VISION-DMD study, which demonstrated vamorolone’s superiority over placebo. In this study, vamorolone met the primary endpoint of Time to Stand (TTSTAND) velocity versus placebo after 24 weeks of treatment and displayed a favorable safety and tolerability profile.
In addition, three open-label studies showcased the drug’s efficacy and safety when administered at doses ranging from 2-6mg/kg/day for up to 30 months. The submission further includes pharmacokinetic data from a study involving healthy adult Chinese volunteers.
Sperogenix and Santhera’s Deal on Vamorolone Paves the Way for New Duchenne Muscular Dystrophy Treatments in China
Under a license agreement between Sperogenix and Santhera signed in January 2022, Sperogenix holds the exclusive rights to develop and market vamorolone for DMD and other rare diseases in China. Once the drug is marketed, Sperogenix will make sales-based milestones and net product sale-based royalty payments to Santhera.
Duchenne Muscular Dystrophy (DMD) is a rare inherited X-chromosome-linked disease, predominantly affecting males. The potential approval of vamorolone in China is a significant step forward in treating DMD, with Sperogenix leading the initiative following a comprehensive data review. The drug has demonstrated superiority over placebo in the VISION-DMD study and has shown promising results in terms of efficacy and safety in three open-label studies. The anticipation now revolves around the NMPA’s approval decision expected in the first quarter of the next year.
Resource: Pharmaceutical Business Review, March 28, 2024
This article has been prepared with the assistance of AI and reviewed by an editor. For more details, please refer to our Terms and Conditions. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author.
