Marinus Pharmaceuticals receives rare paediatric disease designation from FDA for ganaxolone for the treatment of CDKL5 deficiency disorder

Marinus Pharmaceuticals

30 July 2020 - Marinus Pharmaceuticals announced today that the U.S. FDA has granted rare paediatric disease designation for the company’s product candidate ganaxolone for the treatment of CDKL5 deficiency disorder (CDD), a rare refractory form of paediatric epilepsy.

“The data that were used to support the Rare Pediatric Disease Designation comprised an analysis of existing literature as well as unpublished data specific to the CDD population, documenting that the serious or life-threatening manifestations of CDD primarily affect children,” said Joseph Hulihan, M.D., Chief Medical Officer at Marinus. "We plan to publish these new data that will describe important clinical and health economic outcomes related to how seizure burden in children with CDD may affect future developmental milestones."

Read Marinus Pharmaceuticals press release

Michael Wonder

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Michael Wonder