MeiraGTx Ltd, a clinical stage gene therapy company based in London and New York, announced that the US FDA has granted Fast Track designation to their gene therapy product candidate, AAV-RPGR, for the treatment of X-linked retinitis pigmentosa (XLRP). XLRP is one of the most severe forms of retinitis pigmentosa (RP) that leads to blindness, for which there are currently no approved treatments. MeiraGTx is developing AAV-RPGR to target the most common form of XLRP caused by mutations in the eye-specific form of the retinitis pigmentosa GTPase regulator (RPGR) gene, called RPGR-ORF 15. MeiraGTx Ltd is conducting a Phase 1/2 clinical trial of AAV-RPGR in both adult and pediatric XLRP patients with mutations in RPGR-ORF 15. AAV-RPGR has received Orphan Drug designation from the U.S. FDA and Orphan Medicinal Product designation from the European Medicines Agency (EMA).
Fast Track designation is one of five tactics introduced by the Food and Drug Administration (FDA) to make new drugs available as rapidly as possible. Its aim is to aid in the development and accelerate the review of drugs which show promise in treating a serious or life-threatening disease and address an unmet medical need, such as in the case of RP. Now that AAV-RPGR has received Fast Track designation, early and frequent communication between the FDA and MeriaGTx is encouraged throughout the entire process. This increased frequency of communication enables quick resolution of questions and issues, with the aim of earlier drug approval and access by patients.
"XLRP is a devastating condition that causes rapid progression to blindness and currently has no approved treatment options. This Fast Track designation is an important milestone for both patients living with XLRP and MeiraGTx, allowing our team to communicate closely and often with the FDA as we work to bring a much-needed therapy to patients," said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx.
MeiraGTx is currently conducting an open label, Phase 1/2 dose escalation clinical trial of AAV-RPGR in both adult and paediatric patients diagnosed with XLRP caused by RPGR-ORF 15. MeiraGTx is also continuing to enroll an ongoing natural history study of patients with XLRP.
Dr. Alexandria Forbes, MeiraGTx CEO and president, commented: "We look forward to continuing this momentum in our commitment to develop innovative gene therapy treatments for patients."