GC Biopharma received approval from Russia’s health ministry for Hunterase ICV, a treatment for severe Hunter syndrome that delivers medication directly to the brain through an implanted device. The approval makes Russia the second country after Japan to greenlight the therapy.
The treatment addresses a critical gap in care for patients with the rare genetic disorder, which affects about 70% of Hunter syndrome cases. Traditional intravenous therapies often fail to reach the brain due to the blood-brain barrier, limiting their effectiveness against cognitive symptoms.
The company’s Development Division head Lee Jae-woo said the approval builds on Hunterase’s existing presence in Russia, where its intravenous version has been available since 2018.
Hunter syndrome occurs when patients lack an enzyme called iduronate-2-sulfatase, leading to the buildup of complex sugars that damage organs and cognitive function. The new treatment method allows the drug to bypass the blood-brain barrier and directly target affected brain tissue.
GC Biopharma is currently running Phase 1 trials of Hunterase ICV in South Korea as it seeks to expand access to the treatment. The approval represents a key milestone for patients with limited treatment options for the neurological aspects of their condition.
