According to a story from BioSpace, the pharmaceutical company Marinus Pharmaceuticals, Inc. announced recently that its investigational product candidate ganaxolone has earned Rare Pediatric Disease designation from the US Food and Drug Administration (FDA). The drug is being developed as a treatment for CDKL5 deficiency disorder (CDD), a disorder linked to epilepsy in children. Marinus is focused on the development of innovative treatments for rare disorders that cause seizures.
About CDKL5 Deficiency Disorder (CDD)
CDKL5 deficiency disorder is a genetic disorder which is characterized by intellectual disability, seizures, and developmental delays. As the name suggests, the disorder is linked to mutations of the CDKL5 gene which usually occurs spontaneously and is not inherited from a person’s parents. Symptoms become noticeable in the first few months of life. Females are affected more frequently than males, but males tend to have more severe symptoms. These symptoms can include seizures (1-5 per day), constipation, distinctive facial features, reflux, teeth grinding, and problems with feeding and sleeping. Treatment options for CDKL5 deficiency disorder are limited and are primarily focused on minimizing symptoms. Some patients may require a feeding tube. There is a serious need for new and more effective therapies to treat this disorder. In order to learn more about CDKL5 deficiency disorder, click here.
About Rare Pediatric Disease Designation
A medication in development can receive Rare Pediatric Disease designation if it is being developed to treat a condition that is considered rare (defined as any disease that affects less than 200,000 people in the country) and primarily impacts people age 18 or younger. Recipient companies are given a Priority Review voucher once the therapy being developed has been approved for a new drug application (NDA). This voucher can be used for a future application or can be sold to another company. This program is meant to incentivize the development of therapies for rare pediatric diseases.
Ganaxolone is currently being investigated in a phase 3 clinical trial as a treatment for CDLK5 deficiency disorder. It is being developed in both oral and intravenous variants to maximize its effectiveness in both adult and pediatric patients. The medication has the potential to be effective in several different types of difficult-to-treat epilepsy disorders, including tuberous sclerosis complex, status epilepticus, and PCDH19-related epilepsy.