Nacuity Pharmaceuticals Granted U.S. FDA Breakthrough Therapy Designation for NPI-001 (N-acetylcysteine amide) Tablets for the Treatment of Retinitis Pigmentosa
FORT WORTH, Texas, Oct. 02, 2025 (GLOBE NEWSWIRE) -- Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to NPI-001 (N-acetylcysteine amide) tablets, Nacuity’s proprietary, investigational therapy for the treatment of patients with retinitis pigmentosa (RP).
“Breakthrough Therapy Designation represents objective assessment by the FDA that early clinical evidence supports the potential of NPI-001 tablets to deliver substantial treatment effects for patients with retinitis pigmentosa, a serious blinding disease,” said G. Michael Wall, Ph.D., Senior Vice President and Chief Scientific Officer of Nacuity Pharmaceuticals. “This recognition represents a key value-creating milestone for Nacuity and underscores our commitment to efficiently advancing NPI-001 toward late-stage development.”
A drug may receive BTD if the drug is intended to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
In addition to the benefits of Fast Track Designation previously granted to NPI-001, a drug that receives BTD is eligible for intensive guidance on efficient drug development and organizational commitment from FDA.
About Retinitis Pigmentosa
Often diagnosed in childhood or adolescence, retinitis pigmentosa (RP) is a heterogeneous group of inherited retinal diseases involving nearly 3,100 different mutations in more than 50 genes (Daiger et al., 2013), causing progressive night and peripheral vision loss. RP often leads to legal blindness and, in some cases, complete blindness. Forms of RP and related diseases include Usher syndrome, Leber congenital amaurosis and Bardet-Biedl syndrome. According to Foundation Fighting Blindness, an estimated 100,000 people in the U.S. have RP. There are no FDA-approved or standard treatments for RP. A curative gene therapy, voretigene neparvovec (LUXTURNA), is available for patients with the RPE65 mutation, which affects approximately 1-6% of patients with RP (Cross et al., 2022).
About NPI-001
NPI-001 is a proprietary, GMP-grade formulation of N-acetylcysteine amide (NACA) tablets being developed to target oxidative stress associated with diseases such as retinitis pigmentosa (RP). Preclinical studies indicate that NPI-001 boosts glutathione, the body’s most powerful endogenous antioxidant, to stop chemically aggressive oxygen molecules from damaging retinal cells (Wood et al., 2024). NPI-001 is formulated as a stable tablet, easy for patients to self-administer, with well-established manufacturing processes and convenient packaging options. In addition to Breakthrough Therapy Designation, NPI-001 has also been granted Fast Track and Orphan Drug Designations for the treatment of RP, the latter of which provides seven years of U.S. FDA regulatory exclusivity for the product upon regulatory approval.
About Nacuity Pharmaceuticals, Inc.
Nacuity Pharmaceuticals is a clinical-stage leader in innovative treatments for diseases and conditions involving oxidative stress. The company’s powerful, targeted therapies aim to attenuate oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has operations in Fort Worth, TX, USA, and Australia, and extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org). For more information, please visit www.nacuity.com.
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