MeiraGTx Announces AAV-RPGR Granted Fast Track Designation by FDA for Treatment of X-Linked Retinitis Pigmentosa Due to RPGR Deficiency

MeiraGTx Limited, a clinical stage gene therapy company, announced that the 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.

“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,” Alexandria Forbes, PhD, president and chief executive officer of MeiraGTx, said in a company news release. “Marking the third regulatory milestone our ocular program has received this year, we look forward to continuing this momentum in our commitment to develop innovative gene therapy treatments for patients.”

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 (RPGR-ORF 15). AAV-RPGR has received Orphan Drug designation from the FDA and Orphan Medicinal Product designation from the European Medicines Agency (EMA). In addition to the phase 1/2 study that MeiraGTx is conducting, MeiraGTx is also continuing to enroll and conduct an ongoing natural history study of patients with XLRP.

The FDA’s Fast Track process is designed to expedite the development and review of drugs used to treat serious conditions and fill an unmet medical need. Fast Track designation enables the company to have early and frequent communication with the FDA throughout the drug development and review process, which may facilitate faster drug approval and patient access.