The Japan Pharmaceutical and Medical Devices Agency (PMDA) has approved Luxturna for the treatment of inherited retinal dystrophies (IRDs) caused by biallelic RPE65 mutations – Novartis

The Japan Pharmaceutical and Medical Devices Agency (PMDA) has approved Novartis’ Luxturna (voretigene neparvovec) for the treatment of inherited retinal dystrophies (IRDs) caused by biallelic RPE65 mutations.

The company expects the gene therapy to be used in approximately 15 patients in Japan over five years
In the U.S., Luxturna was launched with a list price of $850,000, so Japan is anticipating a high price tag if it gets cleared for reimbursement. It is a one-time administration therapy that works by providing functioning copies of the RPE65 gene to act in place of the mutated RPE65 gene.

The approval was primarily supported by the results of overseas PI (101/102) and PIII (301) studies as well as a Japan PIII trial (A11301) targeting four Japanese patients.