Marinus Pharmaceuticals Earns Rare Pediatric Disease Designation from FDA for Ganaxolone Treating CDKL5 Deficiency Disorder (CDD)

Marinus Pharmaceuticals | July 31, 2020

Marinus Pharmaceuticals Earns Rare Pediatric Disease Designation from FDA for Ganaxolone Treating CDKL5 Deficiency Disorder (CDD)
Marinus Pharmaceuticals, Inc., a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s product candidate ganaxolone for the treatment of CDKL5 deficiency disorder (CDD), a rare refractory form of pediatric epilepsy.

“We are pleased that ganaxolone has received Rare Pediatric Disease Designation from the FDA and are enthusiastic that the timing of this news aligns with our continued expectation to report top line data this quarter from our pivotal Phase 3 Marigold Study evaluating oral ganaxolone in patients with CDD,” said Scott Braunstein, M.D., Chief Executive Officer at Marinus. “This designation for ganaxolone for CDD underscores the significant unmet medical need for children and young adults with this serious and rare genetic disease, which causes early-onset, difficult-to-control seizures and severe neuro-developmental impairment. Our goal is to advance a pipeline of programs that can treat rare seizure disorders, including CDD, tuberous sclerosis complex and PCDH19-related epilepsy, and bring much-needed medicines to patients as soon as possible.”

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Pharma IQ speaks to Stuart Bell, Director of Strategic Services, Idis Managed Access about the challenges and opportunities that stand with early access programmes to medicines.

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Spotlight

Pharma IQ speaks to Stuart Bell, Director of Strategic Services, Idis Managed Access about the challenges and opportunities that stand with early access programmes to medicines.