Key points from article :
The first small-molecule cell regeneration treatment to be developed for the leading cause of inherited blindness.
Endogena Therapeutics announced that the U.S. FDA has granted orphan drug designation for its EA-2353 ophthalmic suspension targeting retinitis pigmentosa.
Retinitis pigmentosa is a rare condition (affects fewer than 200,000 people in the U.S.) that causes slow and progressive loss of vision.
The compound EA-2353 is the first small-molecule being developed for endogenous photoreceptor regeneration.
The treatment works by selective regulation of the endogenous adult stem- and progenitor cells for controlled tissue repair.
The approach is gene-independent and has significant advantages in inherited retina diseases that have multiple genetic causes.
Matthias Steger, CEO of Endogena, said: “Receiving orphan drug designation for EA-2353 is an important milestone ... we anticipate enrolling our first patients into the clinical trial by the end of this year.”
This provides benefits including market exclusivity once approved.
