Switzerland-based Santhera Pharmaceuticals (SWX: SANN) has announced that its Chinese partner Sperogenix Therapeutics has initiated an early access program (EAP) for the rare disease therapy Agamgree (vamorolone) in Hainan’s Bo’ao Lecheng Pilot Zone, with the first patients now receiving treatment for Duchenne muscular dystrophy (DMD). Sperogenix secured the rights to develop and commercialize Agamgree for DMD and other rare disease indications in China under an agreement established in January 2022, which involved an upfront payment of USD 20 million and a commitment to potential milestone payments of USD 104 million. The drug was submitted for approval to China’s National Medical Product Administration (NMPA) in March, with a formal market approval decision anticipated in Q1 2025. The Bo’ao pilot scheme permits drugs that address significant unmet medical needs to be requested for use by patients prior to formal market approval. Hainan’s Medical Products Administration (MPA) approved the availability of Agamgree via the pilot zone in April this year.
Agamgree, already approved for DMD in the US, EU, and other global markets, is an innovative drug that works by binding to the same receptor as glucocorticoids but modulates downstream activity. This molecule has demonstrated the potential to separate efficacy from the safety concerns associated with steroids, offering a new option as a dissociative anti-inflammatory drug and an alternative to the existing corticosteroids, which are the current standard of care for children and adolescent patients with DMD.- Flcube.com
