Marinus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, last week announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ganaxolone for the treatment of CDKL5 Disorder. Ganaxolone is currently being evaluated in children with CDKL5 Disorder in a Phase 2 clinical trial.
“CDKL5 Disorder is a severe, rare genetic disorder that affects children at an early age and causes difficult-to-control seizures and neuro-developmental impairment,” remarked Christopher M. Cashman, Chief Executive Officer of Marinus Pharmaceuticals. “There are no approved therapies for children with CDKL5 Disorder, and a great need for new treatment options that can control both the seizures and co-morbidities of the disease to improve the quality of life for the child and their family. We are pleased to receive Orphan Drug Designation for ganaxolone in CDKL5 Disorder and look forward to presenting the data from our ongoing Phase 2 trial in the upcoming months.”
Orphan Drug Designation is granted by the FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
About CDKL5 Disorder
CDKL5 Disorder is a serious and rare genetic disorder that is caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. It predominantly affects girls and is characterized by early-onset, difficult-to-control seizures and severe neuro‑developmental impairment. The CDKL5 gene encodes a protein essential for normal brain function.
Most children affected by CDKL5 cannot walk, talk, or care for themselves. Many also suffer from scoliosis, visual impairment, gastrointestinal difficulties, and sleeping disorders. Currently, there are no approved therapies for CDKL5 Disorder.