FDA Fast Track Designation for retinitis pigmentosa treatment

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The US Food and Drug Administration (FDA) has designated the investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) as a Fast Track development programme.  

Fast Track is a process designed to enable patients to benefit earlier from important new drugs for serious conditions.  

EA-2353 takes a novel, small-molecule approach and selectively activates endogenous retinal stem and progenitor cells, which differentiate into photoreceptors and can potentially preserve or restore visual function. The drug was granted orphan drug designation by the FDA in May 2021. 

RP consists of a group of inherited diseases causing slow and progressive retinal degeneration and loss of vision, for which there is currently no treatment for most patients. It is a leading cause of inherited blindness, with an estimated 1.5 million people worldwide presently affected. 

Endogena Therapeutics is currently conducting a Phase I/IIa dose-escalation study in collaboration with lead investigator, Mark Pennesi, Professor of Ophthalmology at the Casey Eye Institute in Oregon, USA, to examine the safety, tolerability and preliminary efficacy of EA-2353 administered by intravitreal injection.  

A total of 14 patients with RP due to any pathologic genetic mutation are being recruited across up to six sites in the USA, and the first patient was dosed in July 2022. 

Matthias Steger, CEO of Endogena, said: “This acknowledgement by the FDA of the potential of EA-2353 for RP gives hope for patients living with this devastating degenerative disease. 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 six years to reach this point.” 

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