The Janssen Pharmaceutical Companies of Johnson & Johnson announced that the European Medicines Agency (EMA) has granted both PRIME (PRIority MEdicines) and Advanced Therapy Medicinal Product (ATMP) designations to the company’s adeno-associated virus (AAV)-RPGR gene therapy product for the treatment of inherited retinal disease X-linked retinitis pigmentosa (XLRP).
PRIME is awarded to increase interactions, optimize development plans and accelerate innovative treatments where there is unmet medical need. Similarly, ATMP status is granted to medicines that are based on genes, tissues or cells and can offer groundbreaking opportunities for the treatment of disease. The novel AAV-RPGR asset, which is being jointly developed with MeiraGTx Holdings, also received Fast Track designation from the FDA and orphan designations from the FDA and the EMA.
The PRIME designation is based on data from the ongoing phase 1/2 clinical trial (NCT03252847). For more information, visit https://clinicaltrials.gov/ct2/show/NCT03252847?term=rpgr&draw=2&rank=1.
“The PRIME and ATMP designations for our gene therapy asset are important achievements for Janssen’s growing retinal portfolio and bring us one step closer to delivering a transformational therapy to European patients living with X-linked retinitis pigmentosa,” James List, MD, PhD, Global Therapeutic Area Head, Cardiovascular & Metabolism, Janssen Research & Development, said in a company news release. “We look forward to partnering with the EMA and appreciate their dedication to patients underserved with current options.”
XLRP represents one of the most severe forms of retinitis pigmentosa, resulting in juvenile onset and progression to legal blindness in adulthood. Currently, there are no approved treatments. Janssen’s AAV-RPGR gene therapy is designed to treat the most common form of XLRP, caused by mutations in the RPGR gene, by slowing the retinal degeneration and preserving visual function.
In January 2019, Janssen entered into a worldwide collaboration and license agreement with MeiraGTx Holdings, a clinical-stage gene therapy company, to develop, manufacture and commercialize its inherited retinal disease portfolio. AAV- RPGR gene therapy is being developed under this collaboration and license agreement. The companies also formed a research collaboration to explore new targets for other inherited retinal diseases and further develop AAV manufacturing technology.