Tuesday, November 5, 2024

Tuesday, November 5, 2024

Dravet Syndrome drug submitted for investigation


An investigational new drug application and fast track designation request for EPX-100, a treatment for Dravet Syndrome, were submitted to the Food and Drug Administration.

“This IND filing for EPX-100 is one of the company's significant development milestones. We will test its safety and efficacy in a prompt manner with an eye toward helping many patients who suffer from this disorder,” said Hahn-Jun Lee, president and CEO of Epygenix Therapeutics.

Dravet Syndrome is a rare, lifelong form of epilepsy that is not controlled by current medications. It is caused by heterozygous de novo mutation or deletion of SCN1A, a gene encoding a brain voltage-gated sodium channel.

“We are very pleased to collaborate with Epygenix to support the IND filing for EPX-100, as well as to manage the Phase 1 study in normal volunteers,” said Jules Mitchel, president at Target Health, the lead regulatory and clinical CRO for the EPX-100 development program.

The seizures, which start in the first year of life, can lead to intellectual disability, behavioral abnormalities, gait and motor dysfunction, and increased mortality.

More News

FDA Health News