When Marinus Pharmaceuticals, Inc.’ Ztalmy (ganaxolone) CV was approved by the FDA in March 2022 for seizures associated with a rare, genetic pediatric onset epilepsy disorder, nobody was more elated than CEO Scott Braunstein. Having come out of retirement to advance the development of the positive allosteric modulator of the GABAA receptor – which had previously failed in clinical trials for Fragile X syndrome and post-partum depression – this was a make-or-break moment for the company and the drug.
Ztalmy, an oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder in patients two...