Marinus Ganaxolone nabs Rare Pediatric Disease tag for pediatric epilepsy type

The FDA has granted Rare Pediatric Disease Designation to Marinus Pharmaceuticals’ (MRNS +3.1%) for its product candidate Ganaxolone, an allosteric modulator of GABA_A 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.

https://seekingalpha.com/news/3597738-marinus-pharmas-ganaxolone-nabs-rare-pediatric-disease-tag-for-type-of-pediatric-epilepsy