MeiraGTx (MGTX) Announces PPRIME) and ATMP Designations Granted by EMA to AAV-RPGR Gene Therapy for the Treatment of X-Linked Retinitis Pigmentosa

MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Advanced Therapy Medicinal Product (ATMP) designations to AAV-RPGR, MeiraGTx’s investigational gene therapy for the treatment of x-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.

MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), part of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

PRIME designation was granted based on clinical data from MeiraGTx and Janssen’s ongoing Phase 1/2 trial of AAV-RPGR in patients with XLRP (NCT03252847). AAV-RPGR is the only gene therapy in development for the treatment of XLRP to receive PRIME designation.

“XLRP is a severe disease which causes rapid progression to blindness and total loss of vision in most patients by the fourth decade,” said Alexandria Forbes, Ph.D., president and CEO, MeiraGTx. “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.”

To be eligible for PRIME, medicinal products must be of major public health interest and target conditions where there is an unmet medical need. To determine PRIME eligibility, the EMA considers whether the available data suggest that the product may potentially offer a major therapeutic advantage to patients in a given indication through a clinically meaningful improvement, such as having an impact on the prevention, onset and duration of the condition, or improving the morbidity or mortality of the disease. To be accepted for PRIME, early clinical data must demonstrate the medicine’s potential to benefit patients with unmet medical needs. Features of PRIME designation include guidance and interaction with the EMA on the drug development plan, including early interactions to discuss the regulatory pathway and potential ways to generate data packages designed to address EMA Marketing Authorization Application (MAA) requirements.

ATMP status is granted to medicines that are based on genes, tissues or cells and can offer groundbreaking new opportunities for the treatment of disease. All advanced therapy medicines are authorized centrally via the EMA and may benefit from a single evaluation and authorization procedure.

For more information about the ongoing clinical trial, please visit https://clinicaltrials.gov/ct2/show/NCT03252847.

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