Posted by Michael Wonder on 06 Mar 2019
FDA grants rare paediatric disease designation to odiparcil for the treatment of MPS VI
5 March 2019 - Inventiva eligible to receive priority review voucher upon approval of odiparcil for the treatment of MPS VI.
Inventiva today announced that the U.S. FDA has granted rare paediatric disease designation to odiparcil, the Company's product candidate in development for the treatment of mucopolysaccharidosis (MPS) VI, a type of rare, progressive genetic disorder characterised by a deficiency in the lysosomal enzymes responsible for the normal degradation of glycosaminoglycans.
The designation of rare paediatric disease status confirms odiparcil's eligibility to receive a priority review voucher upon FDA approval of a new drug application to be filed for odiparcil for the treatment of MPS VI.
