FDA grants BTD for Nacuity’s NPI-001 for retinitis pigmentosa

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to NPI-001 (N-acetylcysteine amide) tablets, Texas, USA-based clinical-stage biotech Nacuity Pharmaceuticals’ 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, 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,” he noted.

In 2022, Nacuity licensed rights to Icelandic firm Arctic Therapeutics International for the development and commercialization of NPI-001 in Iceland for the treatment of hereditary cystatin C amyloid angiopathy (HCCAA).