Santhera Pharmaceuticals has received China’s National Medical Products Administration (NMPA) approval for Agamree (vamorolone) to treat patients with Duchenne muscular dystrophy (DMD).
The approval applies to patients aged four years and above in China.
Agamree is already approved for DMD treatment in the US, European Union, and the UK.
This latest approval follows the NMPA acceptance of Agamree’s new drug application (NDA) for DMD in patients aged four and older in March 2024.
The approval highlights the drug’s clinically significant safety benefits, including better bone metabolism, density, and growth compared to standard corticosteroid treatments.
Vamorolone has also been included in the NMPA’s priority review and breakthrough therapy programmes, addressing serious diseases with limited treatment options and offering clear clinical advantages.
Switzerland-based Santhera holds an exclusive global licence from ReveraGen for Agamree, a dissociative steroid with a novel mode of action.
The Swiss speciality drugmaker has out-licensed vamorolone for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics.
Under the $124m licence agreement signed in January 2022, Sperogenix Therapeutics acquired the rights to develop and commercialise Agamree for DMD and other rare disease indications in China.
Santhera will supply vamorolone for the Expanded Access Program (EAP) and commercialisation.
In addition, Sperogenix Therapeutics will pay Santhera double-digit royalties on net product sales, including those from the EAP, along with additional milestone payments based on commercial sales.
Sperogenix Therapeutics co-founder, chairman, and CEO Yan Zhiyu said: “The approval of agamree through the Priority Review Program reflects the government’s high level of attention to the development of rare disease drugs, and also reflects Sperogenix’s firm commitment to the rare disease patients in China who are in urgent unmet needs.”
In the VISION-DMD study, the drug met the primary endpoint, Time to Stand (TTSTAND) velocity, showing significant improvement over placebo at 24 weeks.
It also demonstrated a favourable safety and tolerability profile.
The most common side effects were mild to moderate, including cushingoid features, vomiting, weight gain, and irritability.
Santhera CEO Dario Eklund said: “We are delighted that NMPA has approved Agamree for the treatment of DMD in China and we look forward to working with our partner Sperogenix Therapeutics as it prepares for the commercialisation of the product to ensure DMD patients in China can benefit as soon as possible.”
