The Center for Drug Evaluation (CDE) website has indicated that Merck, Sharp & Dohme’s (MSD; NYSE: MRK) belzutifan and Sperogenix Therapeutics Ltd’s vamorolone are on track to receive priority review statuses in China. Belzutifan, which targets von Hippel Lindau (VHL) disease-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastoma, or pancreatic neuroendocrine tumor (pNET), is already approved in the United States for these indications. Vamorolone, on the other hand, is designed to treat patients aged 4 years and above with Duchenne muscular dystrophy (DMD).
Belzutifan, an oral hypoxia inducible factor (HIF) 2α inhibitor, was first approved in the U.S. in August 2021 for patients with VHL-related RCC, CNS hemangioblastoma, or pNET that require treatment and do not require immediate surgery. In December 2023, it earned an additional indication in the U.S. for the treatment of advanced RCC previously treated with PD-1/L1 inhibitors and VEGF tyrosine kinase inhibitors (TKIs).
Vamorolone, an investigational DMD drug originally developed by Santhera, was licensed to Sperogenix in January 2022 for development and commercialization in Greater China. Vamorolone binds to the same receptor as glucocorticoids but modifies its downstream activity, making it a dissociative anti-inflammatory drug. Its mechanism of action potentially addresses hormone safety issues and could replace glucocorticoids as the standard treatment for DMD patients.
The priority review status for both drugs highlights the potential for faster access to these innovative therapies for patients in China, underscoring the commitment to advancing medical solutions for rare diseases.- Flcube.com
