Nacuity Pharmaceuticals announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation (BTD) to NPI-001 (N-acetylcysteine amide) tablets for treating retinitis pigmentosa (RP), a progressive inherited retinal disease that affects an estimated 100,000 people in the United States. The designation reflects the FDA's assessment that early clinical evidence supports NPI-001's potential to deliver substantial treatment effects for this serious blinding condition.
"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.
Addressing an Unmet Medical Need
Retinitis pigmentosa represents a heterogeneous group of inherited retinal diseases involving nearly 3,100 different mutations in more than 50 genes, causing progressive night and peripheral vision loss. Often diagnosed in childhood or adolescence, RP frequently 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.
Currently, there are no FDA-approved or standard treatments for RP. The only available curative therapy is voretigene neparvovec (LUXTURNA), a gene therapy limited to patients with the RPE65 mutation, which affects approximately 1-6% of patients with RP.
Novel Mechanism of Action
NPI-001 is a proprietary, GMP-grade formulation of N-acetylcysteine amide (NACA) tablets designed to target oxidative stress associated with retinitis pigmentosa. 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.
The drug is formulated as a stable tablet for easy patient self-administration, with well-established manufacturing processes and convenient packaging options. This oral formulation represents a significant advantage in terms of accessibility and patient compliance compared to more complex therapeutic approaches.
Regulatory Pathway and Advantages
The Breakthrough Therapy Designation qualifies NPI-001 for intensive guidance on efficient drug development and organizational commitment from the FDA. A drug may receive BTD if it is intended to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates substantial improvement over existing therapies on clinically significant endpoints.
This designation builds upon NPI-001's existing regulatory advantages. The drug has previously received Fast Track Designation and Orphan Drug Designation for RP treatment. The Orphan Drug Designation provides seven years of U.S. FDA regulatory exclusivity for the product upon regulatory approval.
Company Background
Nacuity Pharmaceuticals is a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress. The company's therapeutic approach focuses on attenuating oxidative tissue damage, which drives blinding eye diseases and a broad spectrum of serious chronic conditions.
The Fort Worth, Texas-based company operates facilities in both the United States and Australia, with backing from Foundation Fighting Blindness and its venture arm RD Fund. Wall emphasized that the breakthrough designation "represents a key value-creating milestone for Nacuity and underscores our commitment to efficiently advancing NPI-001 toward late-stage development."
