FDA grants orphan drug designation to gene therapy for retinitis pigmentosa

AGTC’s gene therapy candidate for X-linked retinitis pigmentosa (XLRP) has been granted orphan drug status by the FDA.

The drug uses an AAV vector to deliver a functional copy of the causative RPGR gene. It previously received an orphan medicinal product designation for the same indication by the European Commission (EC) in June 2016.

“We are on track to file an Investigational New Drug application for this program this year, and this orphan drug designation will help to accelerate the regulatory review process as we work to bring a new treatment option to XLRP patients,” Sue Washer, president and CEO of AGTC.

Additionally, AGTC’s gene therapy candidates for X-linked retinoschisis and achromatopsia have also been granted orphan drug designation from the FDA and EC.