dm+d

Unassigned

New Medicines

CDKL5 deficiency disorder in children and young adults aged 2 to 21 years

Information

New molecular entity
Orion Corporation
Marinus Pharmaceuticals

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Pre-registration (Filed)
Yes
Yes
Aug 21Filed in US for treatment of seizures associated with CDKL5 deficiency disorder; NDA includes a request for Priority Review. Rights to commercialise the oral and IV formulations of ganaxolone in Europe, UK and Switzerland for CDKL5 deficiency disorder, tuberous sclerosis complex and refractory status epilepticus have been transferred to Orion [8,9].
Sep 20Marinus plan to submit a NDA to US FDA mid-2021 and a MAA to EMA for ganaxolone for the treatment of CDD Q3 2021 [6].
Jul 20 FDA grants rare paediatric disease (RPD) designation for ganaxolone for the treatment of CDKL5 deficiency disorder (CDD) [7].
Dec 19Granted orphan drug status in EU [5].
Jun 17Granted orphan drug status in US [3].
EU filing expected by the end of Q3 2021 with plans to launch mid 2022 [10].

Category

GABA A receptor agonist
CDKL5 deficiency disorder (CDD) is cone of most common forms of genetic epilepsy, caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. It is estimated to occur in ~1:40,000 -75,000 live births. Affected individuals experience seizures at a very early age, (usually about 3 months but can be as early as the first week of life) and severe neurodevelopmental delay impacting cognitive, motor, speech, and visual function [1,2].
CDKL5 deficiency disorder in children and young adults aged 2 to 21 years
Oral

Trial or other data

Sep 20PIII NCT03572933 trial (n=101)achieves primary endpoint. Patients receiving oral ganaxolone showed a 32% reduction in 28-day major motor seizure frequency vs. 4% reduction with placebo (p=0.002), at 17 weeks [6].
Jun 18Recruitment to PIII trial NCT03572933 begins [4].

Evidence based evaluations