Posted by Michael Wonder on 07 Sep 2017
Novimmune receives rare paediatric disease designation from the FDA for its lead drug emapalumab
5 September 2017 - Providing eligibility is confirmed at the time of approval of emapalumab for the treatment of primary haemophagocytic lymphohistiocytosis, Novimmune would qualify for a rare paediatric disease priority review voucher.
Novimmune announced today that the US FDA has granted rare paediatric disease designation to its lead compound emapalumab for the treatment of primary haemophagocytic lymphohistiocytosis as a drug for a “rare paediatric disease”.
Priority review vouchers are awarded upon the FDA approval of an eligible drug for a rare paediatric disease. In addition to the Rare paediatric disease designation, emapalumab has orphan drug designation in both the US and Europe, has received FDA’s breakthrough therapy designation, and has been granted eligibility for the European Medicines Agency’s PRIME scheme.
