GRIN Therapeutics Inc, a developer of therapies to treat serious neurodevelopmental disorders, announced on Monday that the US Food and Drug Administration (FDA) has granted Orphan Drug designation for its investigational drug, Radiprodil, intended for the treatment of GRIN-related neurodevelopmental disorder (NDD).

Radiprodil is designed as a potent negative allosteric modulator selectively targeting the N-methyl-D-aspartate receptor subtype 2B (NR2B or GluN2B).

The company is on track to initiate a pivotal Phase 3 trial for radiprodil in mid-2025 for the treatment of GRIN-related NDD with GoF mutations and has an ongoing global open-label clinical trial of radiprodil for the treatment of tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II.

Michael A Panzara, MD, MPH, chief medical officer at Neurvati Neurosciences and GRIN Therapeutics, said: "We are making rapid progress in our efforts to bring a first-ever treatment for GRIN-related neurodevelopmental disorder to patients. Supported by our promising clinical data, the FDA's decision to grant Orphan Drug designation to radiprodil is the latest milestone in that effort. As we plan to launch our pivotal Phase 3 clinical trial to evaluate the impact of radiprodil on seizures, behavioural abnormalities and functional outcomes associated with GRIN-related NDD in the coming months, we are very grateful to the patients and their families who continue to support this important research effort."

Orphan Drug designation is designed to support innovation and research that can lead to more new treatments for diseases that affect fewer than 200,000 people in the United States. The designation grants drug developers the potential for seven years of market exclusivity for their drug after approval, during which time the FDA will generally not approve an application for the same drug targeting the same disease or condition.