FDA grants Rare Pediatric Disease designation for emapalumab

Privately-held Swiss biotech Novimmune has been granted Rare Pediatric Disease Designation to its lead compound emapalumab for the treatment of primary hemophagocytic lymphohistiocytosis (HLH) as a drug for a “rare pediatric disease” by the US Food and Drug Administration.

Priority review vouchers are awarded upon the FDA approval of an eligible drug for a rare pediatric disease. In addition to the Rare Pediatric Disease designation, emapalumab (formerly known as NI-0501) has Orphan Drug designation in both the USA and Europe, has received FDA’s Breakthrough Therapy designation, and has been granted eligibility for the European Medicines Agency’s PRIME scheme.

“We are delighted to have been granted this special designation for our emapalumab program,” said chairman and chief executive Eduard Holdener, adding: “We are looking forward to working with the regulatory authorities to make this medicine available to patients with this devastating disease as soon as possible. This priority review voucher is also precious as it can be used to secure the priority review of another program with the FDA and may be transferred between companies.”