Posted by Michael Wonder on 23 Apr 2018
MeiraGTx announces AAV-RPGR granted fast track designation by U.S. FDA for treatment of X-linked retinitis pigmentosa due to RPGR deficiency
23 April 2018 - MeiraGTx Limited today announced that the U.S. FDA has granted fast track designation for AAV-RPGR for the treatment of X-linked retinitis pigmentosa (XLRP) due to defects in the retinitis pigmentosa GTPase regulator (RPGR) gene.
MeiraGTx is currently conducting an open label, Phase 1/2 dose escalation clinical trial of AAV-RPGR in adult and pediatric patients diagnosed with XLRP caused by mutations in the eye-specific form of the RPGR gene called RPGR open reading frame 15.
AAV-RPGR has received orphan drug designation from the U.S. FDA and Orphan Medicinal Product designation from the EMA. In addition to the Phase 1/2 study that MeiraGTx is conducting, MeiraGTx is also continuing to enrol and conduct an ongoing natural history study of patients with XLRP.
