Nightstar Therapeutics PLC (NASDAQ: NITE) shares saw a massive gain on Thursday after the firm announced a crucial decision from the U.S. Food and Drug Administration (FDA). Essentially, the agency has granted Regenerative Medicine Advanced Therapy (RMAT) designation to NSR-REP1.

For some quick background, NSR-REP1 is the company’s lead product candidate and is currently in Phase 3 development for the treatment of choroideremia, a rare, degenerative, genetic retinal disorder that leads to blindness.

The designation for NSR-REP1 was based on clinical data supporting the maintenance and improvement of visual acuity from completed Phase 1/2 trials

The RMAT designation is an expedited program for the advancement and approval of regenerative medicine products. RMAT allows companies developing regenerative medicine and gene therapies to work more closely and frequently with the FDA, and grants all of the benefits of Breakthrough Therapy Designation, including eligibility for priority review, rolling review and accelerated approval.

Dave Fellows, Chief Executive Officer, commented:

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.

Shares of Nightstar were last seen up about 20% at $16.77, with a consensus analyst price target of $28.50 and a 52-week range of $10.01 to $24.93.

Get Our Free Investment Newsletter

I have read, and agree to the Terms of Use

Thank you for reading! Have some feedback for us?
Contact the 24/7 Wall St. editorial team.