Updated: November 10, 2016
Ganaxolone is a GABA modulator currently being investigated by Marinus Pharmaceuticals. Clinical trials are currently underway to evaluate ganaxolone in the treatment of pediatric genetic orphan epilepsies, behaviors in Fragile X Syndrome, and status epilepticus.
PCDH19 Pediatric Epilepsy: Phase II
PCDH19 pediatric epilepsy is a condition where the patient suffers early-onset cluster seizures. It is typically comorbid with cognitive and behavioral disturbances and may include intellectual disability. The condition is caused by a mutation in the PCDH19 gene. Ganaxolone is currently undergoing an exploratory Phase II clinical trial oral treatment of this condition via liquid suspension and capsules. For PCDH19 pediatric epilepsy, it is expected that ganaxolone will move onto Phase III trials and receive FDA approval in 2021.
There are currently no FDA approved drugs for the treatment of PCDH19 pediatric epilepsy. Anti-seizure medication is sometimes used to help control symptoms. No direct competitors to ganaxolone are currently in development for this condition.
Fragile X Syndrome: Phase II
Fragile X Syndrome (FXS) is an inherited genetic disorder caused by a mutation in the FMR1 gene. Symptoms can include learning disabilities, cognitive impairment, and behavioral issues. A Phase II proof-of-concept trial is underway using ganaxolone in a liquid suspension in an effort to treat symptoms of FXS. For FXS, ganaxolone will likely continue on to Phase III trials and receive FDA approval in 2021.
No drug has yet been approved by the FDA for the treatment of FXS, however several are in development. Metadoxine Extended Release (MDX) is in development by Alcobra Ltd. Results of a Phase II clinical trial have been reported for MDX treating Fragile X. NNZ-2566 is in development by Neuren Pharmaceuticals and has also completed a Phase II clinical trial.
Status Epilepticus: Phase I
Status epilepticus (SE) is a condition where the patient suffers an epileptic seizure with a duration longer than 5 minutes or suffers multiple seizures within this 5 minute period without recovery. Patients who continue to suffer SE even after treatment with antiepileptic drugs (AED) are categorized has having established status epilepticus (ESE). Preclinical studies of intravenous ganaxolone in ESE have yielded positive results. Phase I clinical trials will begin soon - if these clinical trials are successful, ganaxolone is expected to move forward and complete the FDA approval process in 2022.
There are currently no FDA-approved drugs for treating SE. Instead, anti-epileptic drugs such as benzodiazepine, fosphenytoin, and/or phenobarbital. There are no direct competitors to ganaxolone in development as of April 2016.
Managed Care Segment
The managed care segment for ganaxolone is 2B.
Selected Sample of Epidemiology Sources Used in This Analysis
Coffee B, Keith K, Albizua I, et al. Incidence of fragile X syndrome by newborn screening for methylated FMR1 DNA. Am J Hum Genet. 2009;85(4):503-14.
Arrieta I, Criado B, Martinez B, et al. A survey of fragile X syndrome in a sample from Spanish Basque country. Ann Genet. 1999;42(4):197-201.
(See full report for complete source list)
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