Santhera Gets NMPA Approval for AGAMREE (Vamorolone) in Duchenne Muscular Dystrophy

Santhera Pharmaceuticals announces that China’s National Medical Products Administration (NMPA) has approved AGAMREE (vamorolone) for use in China in patients aged 4 years and older.

“DMD is a devastating condition affecting over 70,000 families in China, and until now, there had been no approved treatment option for patients,” said Dario Eklund, CEO of Santhera. “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 commercialization of the product to ensure DMD patients in China can benefit as soon as possible.”

Mr. Yan Zhiyu, Co-founder, Chairman and CEO of Sperogenix Therapeutics, stated: “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. The approval of AGAMREE is an important milestone, and we will continue to uphold our commitment to patient needs and work with stakeholders to accelerate supply and access initiatives, so that more DMD patient families can benefit as soon as possible.”

AGAMREE is the first and only approved therapy for the treatment of DMD in China. This approval follows the acceptance of the new drug application (NDA) filing for AGAMREE in DMD for patients aged 4 years and older by the National Medical Products Administration (NMPA) in March 2024, acknowledging clinically important safety benefits with regards to maintaining normal bone metabolism, density, and growth compared to standard of care corticosteroids. AGAMREE has also been incorporated into both the Priority Review Program and the Breakthrough Therapy Program, which addresses serious diseases lacking effective treatments and includes drugs offering clear clinical advantages over existing treatments.

According to the license agreement between the companies first announced in January 2022, Sperogenix holds exclusive development and commercialization rights to AGAMREE in DMD and all other rare disease indications for China. Santhera is supplying treatment medication to Sperogenix for the EAP as well as for commercialization. Sperogenix will pay Santhera double-digit percentage royalties on net product sales (including for the EAP) and additional revenue-dependent milestones on commercial sales.

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