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news.Marinus Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for ganaxolone, a synthetic analog of the endogenous neurosteroid allopregnanolone, to treat protocadherin 19 gene (PCDH19) female epilepsy.
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Currently, the company is conducting a Phase II trial evaluating the safety and efficacy of ganaxolone as adjunctive therapy for uncontrolled seizures in PCDH19 female pediatric epilepsy, and expects to report initial data later in 2015.
PCDH19 female pediatric epilepsy is a serious and rare epileptic syndrome that is caused by an inherited mutation of the protocadherin 19 (PCDH19) gene, located on the X chromosome.
Around 15,000-30,000 females in the US are affected with this condition, which is characterized by early-onset cluster seizures, cognitive and sensory impairment of varying degrees, and behavioral disturbances.
Allopregnanolone is produced in the central nervous system that modulates the brain neurotransmitter GABA.
Marinus Pharmaceuticals chief executive officer Christopher Cashman said: "We are pleased to receive the Orphan Drug Designation for ganaxolone in PCDH19 female epilepsy.
"This designation underscores the significant unmet medical need for girls suffering from this severe epileptic syndrome, associated with clusters and other types of seizures.
"We believe that the novel mechanism of ganaxolone, along with the established safety profile seen in pediatric epilepsy trials, supports the potential for ganaxolone to control seizures in these young girls who currently have no approved treatment options."
The company is focused on the development of new neuropsychiatric therapeutics and its clinical stage drug candidate, ganaxolone, is designed to treat seizure disorders in adults and children with epilepsy.