According to a story from BioPortfolio, the gene therapy company Nightstar Therapeutics recently released an announcement that the FDA issued Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational product NSR-REP1. This medication is in development for the treatment of choroideremia. As of now, NSR-REP1 is in Phase 3 of clinical trials. This designation indicates the potential that this new gene therapy has demonstrated so far in the treatment of a rare and debilitating disorder.
Choroideremia is a rare genetic disorder that causes degeneration of the retina in affected men. It can lead to blindness if left untreated. Choroideremia progresses slowly, first causing night blindness in childhood, followed by the loss of peripheral and then central vision later in life. The disease is caused by a mutation on the CHM gene. Since the gene is found on the X chromosome, women can be carriers of the mutation but will never experience symptoms. There are no available treatments for choroideremia, and it remains a serious unmet medical need. There are steps that one can take to slow the loss of vision, however. These include the use of UV blocking sunglasses and a diet rich in fresh fruit, leafy vegetables, omega-3 fatty acids, and vitamin supplements. To learn more about choroideremia, click here.
RMAT designation is reserved for regenerative therapies that demonstrate the potential to treat, cure, or reverse a serious, life-threatening disease, particularly if the therapy could address a previously unmet medical need. The designation allows the drug developer to collaborate more closely with the FDA, and also carries all of the same benefits of Breakthrough Therapy designation, such as eligibility for accelerated approval and priority review.
NSR-REP1 could represent a major breakthrough in treatment for choroideremia, and the RMAT designation is based on data gathered in Phase 1 and 2 of clinical trials, in which the treatment was able to cause substantial improvements in vision for study participants with choroideremia.
Hopefully, NSR-REP1 will continue to perform well in its current trials. If it gains approval, it would mean a dramatic improvement in quality of life for patients with this debilitating and degenerative disease.