ProQR Therapeutics NV medicine QR-421a was granted the U.S. Food and Drug Administration's fast-track designation to treat vision loss associated with certain genetic disorders.
The Netherlands-based biopharmaceutical company's drug is being developed to treat vision loss associated with Usher syndrome type 2 and nonsyndromic retinitis pigmentosa caused by mutations in certain genes.
Usher syndrome type 2A is a genetic condition characterized by hearing loss from birth and progressive vision loss that begins in adolescence or adulthood while retinitis pigmentosa is a genetic disorder that begins with night blindness, leading to further vision loss.
ProQR said Usher syndrome, the leading cause of combined deafness and blindness, has no available therapies. The company plans to begin a phase 1/2 trial of the drug and expects preliminary results by mid-year.
The U.S. regulator's fast-track status is granted to drugs being developed for serious conditions that have the potential to fulfill an unmet medical need. The designation provides for an expedited review process for the medicine.