FDA grants fast track designation to N-acetylcysteine amide tablets from Nacuity Pharmaceuticals

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Clinical stage biopharmaceutical company Nacuity Pharmaceuticals announced that the US FDA has granted Fast Track designation for its NPI-001 (N-acetylcysteine amide) tablets, a proprietary investigational therapy for the treatment of patients with retinitis pigmentosa (RP), according to a news release.¹

“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, PhD, senior vice president and chief scientific officer of Nacuity, in the release. “We are committed to advancing NPI-001 to address this significant unmet medical need for patients suffering from RP.”

NP-001 will also be granted Orphan Drug Designation for RP, which provides 7 years of US FDA regulatory exclusivity for the product upon regulatory approval, the release said.

The US FDA will consider a drug for Fast Track designation if it is intended to treat a serious or life-threatening disease and demonstrates a potential to address a currently unmet medical need.¹ Additionally, a fast track drug must show some advantage over available therapy, including the demonstration of superior effectiveness, effect on serious outcomes, or improved effect on serious outcomes, avoiding serious side effects of an available therapy, and ability to address emerging or anticipated public health need, among other factors.²

The tablets are currently being developed to target oxidative stress associated with disease such as RP and has a GMP-grade formulation. Preclinical studies evaluating NPI-001 have found that it boosts the endogenous antioxidant glutathione, which stops chemically aggressive oxygen molecules from further damaging retinal cells.¹

References:

1. Nacuity Pharmaceuticals granted US FDA Fast Track designation for NPI-001 (N-acetylcysteine amide) tablets for the treatment of retinitis pigmentosa. News release. Nacuity Pharmaceuticals. January 21, 2025. Accessed January 21, 2025. https://www.nacuity.com/news/nacuity-pharmaceuticals-granted-u-s-fda-fast-track-designation-for-npi-001-n-acetylcysteine-amide-tablets-for-the-treatment-of-retinitis-pigmentosa/

2. Fast track. US Food and Drug Administration. Accessed January 21, 2025. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track