Project ALS Granted FDA Orphan Drug Designation for Prosetin

Project ALS announced the Food and Drug Administration (FDA) has granted orphan drug designation to prosetin for the treatment of ALS. Prosetin is an oral, brain penetrant MAP4 kinase inhibitor developed by scientists at Columbia University for the treatment of ALS. Project ALS funded prosetin's preclinical development and is sponsoring its translational and potential clinical development.

Orphan status, a designation established by the U.S. Orphan Drug Act of 1983, provides incentives for the development of drugs, biologics, and devices for diseases affecting 200,000 or fewer Americans. By obtaining orphan drug designation, prosetin is now eligible for benefits including up to seven years of marketing exclusivity if it receives regulatory approval, exemption from FDA user fees, FDA assistance in clinical trial design, and tax credits for clinical research.

"Receiving orphan drug designation is an important milestone," said Meredith Estess, president and co-founder of Project ALS. "While prosetin's development is a non-profit effort driven by our commitment to ALS patients, we are proud to oversee this process with the same rigor and diligence as would any drug company."