Nacuity’s NPI-001 investigational mutation-agnostic oral tablet therapy for retinitis pigmentosa has been greenlit for speedier approval by the FDA.
The need for speed has been granted to an intriguing new pill-based oral therapy for retinitis pigmentosa (RP).
NPI-001 (Nacuity Pharmaceutical; Texas, USA) is a proprietary formulation of N-acetylcysteine amide (NACA), designed to combat oxidative stress—a key driver of retinal cell damage in RP. Preclinical studies suggest that NPI-001 boosts glutathione, the body’s primary antioxidant, helping to protect retinal cells from further damage.

[Source: Nacuity Pharmaceuticals]
Fast Track designation is reserved for therapies targeting serious conditions with unmet medical needs. It enables more frequent interactions with the United States Food and Drug Administration (FDA), potentially paving the way for Accelerated Approval or Priority Review, should the treatment meet additional criteria.
“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,” said G. Michael Wall, senior vice president and chief scientific officer of Nacuity, in a news release. “We are committed to advancing NPI-001 to address this significant unmet medical need for patients suffering from RP.”
With Fast Track status, Nacuity aims to expedite the development and review process for NPI-001, potentially offering a gene-agnostic treatment option for RP. In addition to its Fast Track designation, NPI-001 holds Orphan Drug Designation, granting it seven years of U.S. market exclusivity upon FDA approval.
RP is an inherited retinal disease characterized by progressive loss of night and peripheral vision. Often diagnosed in childhood or adolescence, it can lead to legal blindness and, in some cases, total vision loss. The condition involves mutations in over 50 genes, with around 100,000 people in the United States affected.
Currently, voretigene neparvovec (Luxturna®; Spark Therapeutics; Pennsylvania, USA) is the only FDA-approved treatment available, but it is specific to patients with mutations in the RPE65 gene, representing only a small fraction of RP cases. For the broader population, no FDA-approved therapies exist.
This FDA designation shines a hopeful light on a future where more RP patients might preserve their vision—and their quality of life. Stay tuned for updates as this novel therapy continues its journey through clinical development.