Cellusion Receives FDA Orphan Drug Designation for CLS001

Cellusion announced that the FDA has granted orphan drug designation to its regenerative medicine product, “iPS cell-derived corneal endothelial cell substitute,” (CLS001) for the treatment of bullous keratopathy.

FDA grants orphan drug designation if the drug meets specific criteria, the treatment, diagnosis, or prevention of rare diseases that affect less than 200,000 people in the United States. The designation qualifies sponsors for incentives, including tax credits for qualified clinical (in humans) testing, waiver of the prescription drug user fee, and potential 7 years of market exclusivity after FDA approval.

Cellusion has developed CLS001 for bullous keratopathy, which accounts for more than half of all corneal transplants and is currently preparing for corporate clinical trials in Japan and global clinical trials. With this designation, the company said it will further accelerate the global development of CLS001 and aim to contribute to patients around the world who are waiting for the treatment.