Gene Therapy, Pharma

Nightstar’s drug for rare vision loss admitted to FDA’s gene therapy speedway

In the latest show of regulatory support for gene therapies, Oxford spinout Nightstar Therapeutics just got access to a fairly new “fast-lane” to speed its retinal gene therapy to market.

The London-based company scored an FDA designation called Regenerative Medicine Advanced Therapy, or RMAT, for its Phase III drug NSR-REP1. The therapy is designed to treat choroideremia, a rare genetic condition that causes progressive vision loss, by correcting mutations in the CHM gene.

David Fellows

“Receiving RMAT designation for NSR-REP1 highlights the potential of this gene therapy to maintain and improve visual acuity in choroideremia,” said Dave Fellows, Nightstar’s CEO, in a statement. “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.”

RMAT includes all the benefits of the fast track and breakthrough designations, including early interactions between the FDA and sponsors. Back when RMAT was first created (as part of the 21st Century Cures Act), drugmakers worried gene therapies would be excluded. That’s because the FDA didn’t explicitly include gene therapies in the first description of the designation. The agency remedied that, however, last November.

Unlike the breakthrough designation, RMAT doesn’t require evidence that the drug may offer a substantial improvement over available therapies. And like breakthrough designations, getting an RMAT designation doesn’t mean the product will be approved and does not change the standards for safety and effectiveness needed for approval.

RMAT designation for NSR-REP1 was based on clinical data supporting the maintenance and improvement of visual acuity from completed Phase I/II trials in choroideremia patients, the company said. The drug is now in Phase III trials.

Nightstar’s stock $NITE, which first listed on Nasdaq last September, is up nearly 6% on the news.


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Recursion Pharmaceuticals Salt Lake City, UT
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Atlas Venture Cambridge, MA

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