The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to iQ-007, a molecule developed at the Faculty of Pharmacy of the Jagiellonian University Medical College. The drug is intended for the treatment of Dravet Syndrome, a rare and severe form of childhood epilepsy.

iQ-007 is a first-in-class antiepileptic drug candidate that works by removing excess glutamate from the brain. It was developed by the team of Prof. Krzysztof Kamiński.

Orphan Drug Designation expedites the development and registration of drugs for rare diseases. For iQ-007, this means:

Scientific advice from the FDA in clinical trial planning
Faster registration procedures
Extended patent protection
Market exclusivity in the U.S.
Dravet Syndrome is a rare genetic neurological disorder characterized by severe, drug-resistant seizures in young children. iQ-007 offers hope for a more effective and better-tolerated treatment for this condition.

The unique mechanism of action of iQ-007 may also have applications in the treatment of other neurological diseases, such as Alzheimer’s disease, Parkinson’s disease, and multiple sclerosis.

The iQ-007 molecule was commercialized by the Jagiellonian University in 2020, and the license for its development was granted to the American biotechnology company iQure Pharma Inc.