China-based rare disease specialist Sperogenix Therapeutics Limited has announced receiving marketing approval from the National Medical Products Administration (NMPA) for its drug Agamree (vamorolone), which is used to treat patients with Duchenne muscular dystrophy (DMD). This marks a significant milestone as Agamree becomes the first DMD drug approved in China, having previously been awarded breakthrough therapy designation (BTD) and priority review statuses by the NMPA.
Understanding Duchenne Muscular Dystrophy (DMD)
DMD is a rare X-linked recessive genetic disease caused by mutations in the muscle dystrophin gene, leading to the loss of muscle dystrophin, which is essential for maintaining muscle cell membrane stability. The condition results in progressive muscle degeneration and weakness, affecting primarily boys and young men.
Agamree’s Clinical Safety Advantages
Agamree is a new type of dissociative corticosteroid that offers therapeutic effects comparable to traditional corticosteroid drugs but with important clinical safety advantages. It helps maintain normal bone metabolism, bone density, and growth, which are critical factors in the treatment of DMD patients.
Origin and Licensing Deal
Agamree originated with Swiss firm Santhera Pharmaceuticals (SWX: SANN) and is registered in the US and European Union (EU) for the treatment of DMD. Sperogenix acquired the rights to the drug in January 2022 through a licensing deal with Santhera, which involved a USD 20 million upfront payment and potential milestone payments of USD 104 million. The drug received approval in Hainan in April this year through the “first pilot, first trial” policy of the Boao Lecheng Medical Tourism Pilot Zone.-Fineline Info & Tech
