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Nightstar Therapeutics, a UK-based clinical stage gene therapy company, receives US FDA Regenerative Medicine Advanced Therapy (RMAT) designation

Nightstar Therapeutics (NASDAQ:NITE), originally a UK based spin-out of the University of Oxford, based on the founding work of Professor Robert MacLaren at the Nuffield Laboratory of Ophthalmology, has announced the receipt of Regenerative Medicine Advanced Therapy (RMAT) designation from the US FDA.  According to Nightstar, RMAT designation, established under the 21st Century Cures Act, is an expedited program for the advancement and approval of regenerative medicine products benefitting the sponsors of treatments for serious or life-threatening diseases through closer and more frequent interaction with the FDA, including eligibility for priority review and accelerated approval. From a commercial perspective the engagement is hoped to reduce risks and timelines in bringing innovative products to market.


Nightstar’s RMAT designation from the FDA is for the development of NSR-REP1, the company’s lead product candidate currently in Phase III development for the treatment of choroideremia. Choroideremia is a rare X-linked disease caused by mutations in the CHM gene, a transcript encoding the REP1 protein which functions in intracellular protein trafficking and waste disposal in retinal cells.  The disorder afflicts approximately 1 in 50,000 people primarily affecting male patients and is usually diagnosed in childhood.  Dysfunctional REP1 often leads to cell apoptosis causing progressive vision loss and blindness from an early age making the condition a devastating long-term illness.  NSR-REP1 is the company’s lead clinical-stage product comprised of an AAV2 vector housing a functional cDNA for the production of REP1 inside the eye, designed to rescue the cells from the primary genetic lesion.   In preliminary studies of  32 patients treated with NSR-REP1 across four open-label Phase I / II clinical trials, 90%+ of treated patients maintained visual acuity over a two-year follow-up period.


Commenting on the achievement, Dave Fellows, Chief Executive Officer of Nightstar said that receiving RMAT designation for NSR-REP1 “highlights the potential of this gene therapy to maintain and improve visual acuity in choroideremia.  This designation further underscores a recognition of the serious nature of choroideremia and the urgent need to develop new treatments for those affected by inherited retinal diseases that would otherwise lead to blindness. We look forward to working closely with the FDA to discuss the NSR-REP1 development program and to determine how we can accelerate the pathway for making NSR-REP1 available to choroideremia patients.”