The FDA has granted Rare Pediatric Disease Designation to Marinus Pharmaceuticals’ (MRNS +3.1%) for its product candidate ganaxolone, an allosteric modulator of GABAA receptors, for CDKL5 deficiency disorder (CDD), a rare refractory form of pediatric epilepsy.

CDD is characterized by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment.

Rare Pediatric Disease Designation provides for the issuance of a priority review voucher following FDA approval. The voucher can be used for accelerated approval of future marketing application or it can be sold to a third party.


Source link