X-linked retinitis pigmentosa gene therapy granted PRIME, ATMP designations by EMA

The European Medicines Agency has granted priority medicines and advanced therapy medicinal product designations for AAV-RPGR, an investigational gene therapy treatment for X-linked retinitis pigmentosa, according to press releases from MeiraGTx Holdings and Janssen Pharmaceuticals, which are jointly developing the therapy.

The therapy is the only RPGR gene therapy program to be awarded priority medicines (PRIME) designation, which is given “to increase interactions, optimize development plans and accelerate innovative treatments where there is unmet medical need,” according to the Janssen release.

Advanced therapy medicinal product (ATMP) designation is granted to cell-, tissue- or gene-based medicines that provide “groundbreaking” opportunities for disease treatment.

“XLRP is a severe disease which causes rapid progression to blindness and total loss of vision in most patients by the fourth decade,” Alexandria Forbes, PhD, president and CEO of MeiraGTx, said in the company’s release. “People suffering from this devastating disease are currently living without treatment options, and we’re pleased the EMA has recognized the potential this investigational gene therapy brings to patients. We look forward to working closely with the EMA, patient and clinical communities, and our partner Janssen to potentially accelerate the clinical development of AAV-RPGR and bring a much-needed therapy to people affected by XLRP.”

AAV-RPGR previously received fast track designation from the FDA and orphan drug designations from the FDA and EMA.

The European Medicines Agency has granted priority medicines and advanced therapy medicinal product designations for AAV-RPGR, an investigational gene therapy treatment for X-linked retinitis pigmentosa, according to press releases from MeiraGTx Holdings and Janssen Pharmaceuticals, which are jointly developing the therapy.

The therapy is the only RPGR gene therapy program to be awarded priority medicines (PRIME) designation, which is given “to increase interactions, optimize development plans and accelerate innovative treatments where there is unmet medical need,” according to the Janssen release.

Advanced therapy medicinal product (ATMP) designation is granted to cell-, tissue- or gene-based medicines that provide “groundbreaking” opportunities for disease treatment.

“XLRP is a severe disease which causes rapid progression to blindness and total loss of vision in most patients by the fourth decade,” Alexandria Forbes, PhD, president and CEO of MeiraGTx, said in the company’s release. “People suffering from this devastating disease are currently living without treatment options, and we’re pleased the EMA has recognized the potential this investigational gene therapy brings to patients. We look forward to working closely with the EMA, patient and clinical communities, and our partner Janssen to potentially accelerate the clinical development of AAV-RPGR and bring a much-needed therapy to people affected by XLRP.”

AAV-RPGR previously received fast track designation from the FDA and orphan drug designations from the FDA and EMA.