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news.The US Food and Drug Administration (FDA) has granted fast track designation to Nacuity Pharmaceuticals’ NPI-001 (N-acetylcysteine amide) tablets for treating individuals with retinitis pigmentosa (RP).
The fast track status is granted to drugs which show potential in treating a serious or life-threatening condition. Credit: Myriam Zilles on Unsplash.
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Designed to target oxidative stress related to RP, NPI-001 is the company’s investigational candidate.
In preclinical studies, the therapy improved glutathione, which is said to be the most powerful endogenous antioxidant in the body to protect retinal cells from the damage caused by the chemically aggressive oxygen molecules.
The therapy, along with other small molecules, has demonstrated antioxidant activity in various preclinical models, with potential for further applications.
Nacuity Pharmaceuticals chief scientific officer and senior vice-president Michael Wall said: “Fast track designation represents an objective assessment by the FDA for the potential of NPI-001 tablets as a treatment for RP, a severe blinding disease.
“We are committed to advancing NPI-001 to address this significant unmet medical need for patients suffering from RP.”
In addition to the fast track status, the therapy has received orphan drug designation for the same condition.
The fast track status is granted to drugs which show potential in treating a serious or life-threatening condition. Additionally, the programme offers the possibility of accelerated approval and priority review, depending on the fulfilment of specific criteria.
Diagnosed frequently in childhood or adolescence, RP is a complex group of retinal conditions that are inherited, with more than 3,000 mutations in over 50 genes, leading to progressive loss of night and peripheral vision.
RP can lead to legal blindness and, in some instances, complete blindness. It encompasses related conditions such as Leber congenital amaurosis, Bardet-Biedl syndrome, and Usher syndrome.
It is estimated that 100,000 individuals in the country are affected by this eye condition, for which there are no FDA-approved or standard treatments available.
The company noted that its researchers are working on a gene-agnostic treatment for this condition.
