FDA Grants Breakthrough Therapy Designation to Nacuity’s NPI-001 for Retinitis Pigmentosa
Nacuity Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to NPI-001, the company’s investigational oral therapy for retinitis pigmentosa (RP). The designation recognizes early clinical evidence suggesting that NPI-001 may offer substantial benefit to patients with this progressive, inherited retinal condition.
A Novel Approach Targeting Oxidative Stress
NPI-001 is a GMP-grade formulation of N-acetylcysteine amide, designed to mitigate oxidative stress, a key factor contributing to photoreceptor degeneration in RP. According to Nacuity, preclinical data demonstrate that the therapy enhances glutathione levels, helping neutralize reactive oxygen species and protect retinal cells.
“Breakthrough Therapy Designation represents an 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, PhD, 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.”
Clinical Trial Results: Slowed Photoreceptor Loss
The designation follows results from a phase 1/2 randomized, placebo-controlled, double-masked, multicenter clinical trial (NCT04355689) involving 49 patients with RP across four sites in Australia. Participants received oral NPI-001 or placebo tablets twice daily for two years.
Key findings included:
• More than 50% slower photoreceptor loss in the NPI-001 group compared to placebo over 24 months.
• While retinal sensitivity did not reach statistical significance, patients receiving NPI-001 experienced nearly 30% slower loss of visual function.
Regulatory Designations
NPI-001 has also received Fast Track Designation, granted in early 2025, as well as Orphan Drug Designation, which provides seven years of U.S. regulatory exclusivity upon approval.
