Saturday, November 23, 2024

Saturday, November 23, 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.

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