China NMPA approves Santhera's DMD therapy

Santhera Pharmaceuticals has achieved a significant milestone with the approval of its drug AGAMREE (vamorolone) by China’s National Medical Products Administration (NMPA). This medication is designed to treat Duchenne muscular dystrophy (DMD) in individuals aged four years and above, becoming the first and only treatment approved for this purpose in China.

The approval comes following the NMPA’s acceptance of the drug’s new application in March 2024. AGAMREE is noted for its clinically significant safety benefits over traditional corticosteroids, particularly in maintaining normal bone metabolism, density, and growth. The drug has also been fast-tracked through China’s breakthrough therapy and priority review programs, underscoring its potential impact.

Sperogenix, a key partner in the endeavor, holds exclusive rights to develop and commercialize AGAMREE for DMD and other rare diseases within China. This partnership, formed under a licensing agreement in January 2022, has been instrumental in bringing the drug to market. Yan Zhiyu, co-founder, chairman, and CEO of Sperogenix, emphasized the importance of this approval, highlighting the government's focus on rare disease drugs and Sperogenix's dedication to addressing urgent unmet medical needs in China.

Santhera Pharmaceuticals will supply the medication to Sperogenix for both expanded access programs and commercial sales. The agreement entails Santhera receiving double-digit percentage royalties on net product sales, including those from the expanded access program, along with additional revenue-dependent milestones.

The significance of AGAMREE’s approval cannot be overstated, given the substantial impact of DMD, a devastating condition that affects over 70,000 families in China alone. Prior to this, no approved treatment options were available for DMD patients in the country. Santhera CEO Dario Eklund expressed his satisfaction with the NMPA’s decision, looking forward to collaborating with Sperogenix Therapeutics in the product’s commercialization to ensure patients in China gain access to this novel treatment swiftly.

AGAMREE’s efficacy and safety were demonstrated in the pivotal VISION-DMD study, where the therapy met its primary endpoint of time-to-stand velocity compared to a placebo over a 24-week treatment period. The study also confirmed the drug's favorable safety and tolerability profile, positioning it as a promising treatment option for DMD patients.

In addition to its approval in China, AGAMREE was recommended by the National Institute for Health and Care Excellence after a new agreement that includes a discount on the therapy’s price, further validating its therapeutic value.

This approval marks a significant step forward in the treatment of DMD, providing hope and a new therapeutic option for thousands of patients and their families in China. The collaboration between Santhera Pharmaceuticals and Sperogenix Therapeutics underscores the importance of strategic partnerships in advancing healthcare solutions for rare diseases on a global scale.