FDA Grants Orphan Designation to Rare Seizure Disorder Drug


CDKL5 Disorder is characterized by seizures and neuro-developmental impairment.

Marinus Pharmaceuticals recently announced that the FDA granted orphan drug designation to ganaloxone for patients with CDKL5 Disorder, a rare seizure condition, according to a press release.

Ganaxolone is a central nervous system-selective GABAA modulator that is being developed as an intravenous solution, a capsule, and a liquid. Unlike other seizure treatments, ganaxolone displays anti-seizure and anti-anxiety activity by modifying synaptic and extrasynaptic GABAA receptors, according to the release.

To date, the drug has been explored in more than 1500 pediatric and adult patients for up to 2 years and has been deemed safe and well-tolerated. Marinus Pharmaceuticals reported that the most common adverse events included drowsiness, dizziness, and fatigue.

CDKL5 Disorder is a serious and rare genetic condition that is the result of a cyclin-dependent kinase-like 5 (CDKL5) gene mutation. The condition predominantly affects girls and is characterized by seizures and significant neuro-developmental impairment, according to the release.

Most children with the condition cannot walk, talk, or carry out day-to-day activities. These patients may also have scoliosis, visual deficiencies, gastrointestinal problems, and sleeping disorders, according to the release.

Ganaxolone is currently being assessed in these patients 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," Christopher M. Cashman, chief executive officer of Marinus Pharmaceuticals, said in a press release. "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."

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