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# Orphan Status for Marinus Pharmaceuticals’ Ganaxolone
- URL: https://www.fdaweb.com/orphan-status-for-marinus-pharmaceuticals-ganaxolone/
- Published: 2017-06-29T12:00:00.000Z
- Updated: 2026-09-14T22:32:16.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139067

FDA has granted Marinus Pharmaceuticals an orphan drug designation for ganaxolone for treating CDKL5 Disorder. The drug is currently being evaluated in a Phase 2 clinical trial in children with CDKL5 Disorder, which affects children at an early age and causes difficult-to-control seizures and neuro-developmental impairment. Ganaxolone, a positive allosteric modulator of GABAA, is being developed in three different dose forms (intravenous, capsule, and liquid), according to Marinus. “Unlike benzodiazepines, ganaxolone exhibits anti-seizure and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors,” it says.