China’s National Medical Products Administration (NMPA) has approved Santhera Pharmaceuticals’ AGAMREE (vamorolone), used to treat Duchenne muscular dystrophy (DMD) in patients aged four years and above.
It is the first and only approved treatment in the country for this indication.
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The approval follows the NMPA’s acceptance of the new drug application (NDA) in March 2024 which highlighted the therapy’s clinically important safety benefits over standard corticosteroids, including the maintenance of normal bone metabolism, density and growth.
It has also been included in the breakthrough therapy programme and the priority review programme.
Sperogenix holds exclusive rights to develop and commercialise AGAMREE for DMD and other rare diseases in China, under a licensing agreement initially announced in January 2022.
Sperogenix co-founder, chairman and CEO Yan Zhiyu stated: “The approval of AGAMREE through the priority review programme 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 need.”
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By GlobalDataSanthera will supply Sperogenix with the medication for both the expanded access programme (EAP) and commercialisation and is set to receive double-digit percentage royalties on net product sales, including those from the EAP, as well as additional revenue-dependent milestones.
Santhera Pharmaceuticals CEO Dario Eklund stated: “DMD is a devastating condition affecting over 70,000 families in China, and until now, there had been no approved treatment option for patients.
“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.”
In the pivotal VISION-DMD study, the therapy met its primary endpoint time-to-stand velocity, compared to the placebo at a 24-week treatment period, with a favourable safety and tolerability profile.
Before this announcement, the National Institute for Health and Care Excellence recommended the therapy for DMD following a new agreement that includes a discount on the therapy’s price.
