Nacuity Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts, and other diseases caused by oxidative stress. On January 21, 2025, they announced that its investigative therapy for the treatment of retinitis pigmentosa (RP), NPI-001, has received both Fast Track Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).
NPI-001 is a unique form of N-acetylcysteine amide (NACA) tablets that help reduce oxidative stress, which is linked to RP and other diseases. Oxidative stress happens when there is an imbalance or too many naturally occurring free radicals in the body. In small amounts, free radicals can be beneficial; however, when they accumulate, they can harm our cells and contribute to disease progression.
Fast Track Designation is given to potential treatments that address serious or life-threatening conditions and meet unmet medical needs. This means that NP-001 can be reviewed faster and might get approved quicker, so it can be available to patients sooner.
Orphan Drug Designation is awarded to drug candidates targeting rare diseases. This designation provides several benefits, like seven years of exclusive rights to sell the drug in the U.S., possible tax credits for eligible clinical trials, waivers for certain regulatory fees, and support to speed up the development process.
What this means for Usher syndrome: Currently, there is no FDA-approved treatment for all forms of RP. The only approved therapy for inherited retinal diseases (IRDs) is voretigene neparvovec (Luxturna), which is specifically for patients with RPE65 gene mutations. Nacuity’s NPI-001 gene-agnostic treatment has the potential to address a significant unmet medical need for all patients with RP, including Usher syndrome, no matter what their genetic mutations are.
