INTEPLA (Fenfluramine) Receives Orphan Drug Designation in Japan

Release Date: 12-Sep-2021

Zogenix, a global biopharmaceutical company announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation (ODD) to FINTEPLAandreg; (fenfluramine) oral solution to treat Dravet syndrome. Dravet syndrome is a rare, devastating and life-long form of epilepsy that normally begins in infancy and is marked by frequent, treatment-resistant seizures, motor and behavioral impairments, significant developmental, and an increased risk of sudden unexpected death in epilepsy (SUDEP).


FINTEPLA possesses dual activities to inhibit seizures: as a serotonergic agent, acting as a potent 5-HT releaser with agonist activity at 5-HT1D, 2A, and 2C receptors, and as a positive modulator of Sigma1R. FINTEPLA is a prescription medicine used to cure the seizures associated with Dravet syndrome in patients 2 years of age and older. Fenfluramine is approved in Europe and US and is in development in Japan for the treatment of seizures associated with Dravet syndrome.


Based on the Ministry of Health Labour and Welfare (MHLW) Patient Survey there are an estimated 3,000 Dravet syndrome patients in Japan. The incidence of Dravet syndrome in Japan is estimated to be 1 in 20,000-40,000. Affecting one in 15,700 live births in the U.S. and around one in 20,000 to 40,000 live births in Europe, most patients follow a course of developmental delay with cognitive, motor and behavioral deficits that persist into adulthood.

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