The Center for Drug Evaluation (CDE) website indicates that two drugs have been selected for priority review status. The selected products are French giant Sanofi’s Nexviazyme (avalglucosidase alfa) and China-based Hong Kong WinHealth Pharma Group Ltd’s Ravicti (glycerol phenylbutyrate).
Nexviazyme Details
Nexviazyme is an investigational enzyme replacement therapy (ERT) developed by Sanofi for patients with Pompe disease (acid alpha glucosidase [GAA] deficiency). The therapy specifically targets the mannose-6-phosphate (M6P) receptor to increase cellular uptake of GAA and remove glycogen accumulation in muscle cells. The drug has received breakthrough therapy designation (BTD) and priority review status in the US, and was approved in August of last year to treat patients 1 year and older with late-onset Pompe disease.
Ravicti Details
Ravicti is a nitrogen-binding agent used for long-term treatment of patients with urea cycle disorders (UCDs) that cannot be controlled by protein restriction and/or amino acid supplementation alone. The product, originated by Horizon Pharma, was fast-tracked in the US in 2013 and approved for marketing in the EU in 2015. WinHealth Pharma acquired exclusive commercialization rights to the drug in Greater China, Korea, Singapore, Vietnam, Indonesia, Malaysia, Philippines, and Thailand in 2021.-Fineline Info & Tech
