Posted by Michael Wonder on 15 Nov 2017
FDA approves treatment for rare genetic enzyme disorder
15 November 2017 - The U.S. FDA today approved Mepsevii (vestronidase alfa-vjbk) to treat pediatric and adult patients with an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. MPS VII is an extremely rare, progressive condition that affects most tissues and organs.
The FDA granted this application fast track designation, which seeks to expedite the development and review of drugs that are intended to treat serious conditions where initial evidence showed the potential to address an unmet medical need. Mepsevii also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The sponsor is receiving a rare paediatric disease priority review voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare paediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive priority review of a subsequent marketing application for a different product. This is the twelfth rare paediatric disease priority review voucher issued by the FDA since the program began.
