GTX-102 Granted Fast Track Designation by U.S. FDA
GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome (AS). GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of AS.
The FDA’s Fast Track designation is a program designed to facilitate the development and expedite the review of drugs intended to treat serious conditions that demonstrate the potential to address an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process, and allows for a rolling review of a company’s New Drug Application (NDA). Products with Fast Track designation may also be eligible for Priority Review, if relevant criteria are met at the time of NDA filing.