Santhera announces launch of early access program in China for Agamree by its partner Sperogenix,

In April 2024, the Hainan Medical Products Administration (HMPA) authorized the EAP for Agamree based on local policies, Agamree's existing overseas approvals (U.S., EU, UK) and its demonstrated ability to address urgent clinical needs in DMD, where approved treatments are currently unavailable in China. The EAP has started in the Bo’ao Lecheng Pilot Zone, located in Hainan Province, in mid-May, when the first patients were treated with Agamree.

In March 2024, the National Medical Products Administration (NMPA) accepted the new drug application (NDA) filing for Agamree in DMD for patients aged 4 years and older, incorporating it into both the Priority Review Program and the Breakthrough Therapy Program. Subject to a positive review outcome, approval could be obtained by Q1-2025.

Duchenne muscular dystrophy is a rare neuromuscular disease affecting about 70,000 patients in China. Currently, there is no approved drug to treat DMD in China, leaving a high unmet medical need and therapeutic gap, especially considering the increasing diagnosis rates that enable more patients to access specialized treatment centers.

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.