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news.Santhera Pharmaceuticals has reported that the China National Medical Products Administration (NMPA) granted priority review for a new drug application (NDA) for vamorolone, for Duchenne muscular dystrophy (DMD).
A decision on the approval for vamorolone to treat DMD patients aged four years and above is anticipated next year. Credit: Annie Spratt on Unsplash.
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Sperogenix Therapeutics, the company’s rare disease partner in China, filed the application with the NMPA.
The Center for Drug Evaluation (CDE) of the NMPA accepted the filing after previously including vamorolone in the breakthrough therapy programme.
A decision on the approval for the use of vamorolone in DMD patients aged four years and above is anticipated in the first quarter of next year.
The NDA in China is based on a comprehensive data package that has already led to vamorolone’s approval in the US, EU, and UK.
The data supporting the NDA includes results from the VISION-DMD study, which demonstrated vamorolone’s superiority over placebo.
In this study, the therapy met the primary endpoint of Time to Stand (TTSTAND) velocity versus placebo at 24 weeks of treatment. It also exhibited a favourable safety and tolerability profile in the trial.
Additionally, three open-label studies showed the drug’s efficacy and safety when administered at doses ranging between 2-6mg/kg/day for up to 30 months.
The submission also encompasses pharmacokinetic data from a study involving healthy adult Chinese volunteers.
Under a licence agreement between Sperogenix and Santhera signed in January 2022, Sperogenix holds the sole rights to develop and market vamorolone for DMD and other rare diseases in China.
Upon the marketing of the drug, Sperogenix will make sales-based milestone and net product sale based royalty payments to Santhera.
A rare inherited X-chromosome-linked ailment, DMD mostly affects men.
