Feb 9, 2023

FDA grants fast track designation for retinitis pigmentosa treatment

EA-2353 for the treatment of retinitis pigmentosa received fast-track designation from the FDA, according to a press release from Endogena Therapeutics.

The gene-independent treatment approach activates endogenous retinal stem and progenitor cells, which differentiate into photoreceptors to possibly preserve or restore visual function, the release said.

A phase 1/2a dose-escalation study is underway to investigate the safety, tolerability, and preliminary efficacy of EA-2353 administered intravitreally. Fourteen participants with retinitis pigmentosa are being recruited at six U.S. sites, with the first participant dosed in July 2022.

The designation will allow for more rapid regulatory review of a future new drug application, according to the release.

“This acknowledgment by the FDA of the potential of EA-2353 for RP gives hope for patients living with this devastating degenerative disease,” Matthias Steger, PhD, MBA, CEO of Endogena, said in the release. “It is a significant milestone for our company, our investors, and gives recognition to our dedicated team at Endogena, who have been working for the past 6 years to reach this point.”

The FDA previously granted orphan drug designation for EA-2353 in May 2021.

Source: Healio & FDA

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