RARE Daily

FDA Grants Caeregen Rare Pediatric Disease Designation for Rare Eye Disease Therapy

December 9, 2022

The U.S. Food and Drug Administration granted Caeregen Therapeutics Rare Pediatric Disease designation for Noregen, its experimental therapy for treatment of Familial Exudative Vitreoretinopathy, a rare genetic disease of the eye.

Photo: Walter Capone, CEO of Caeregen Therapeutics

Familial Exudative Vitreoretinopathy (FEVR) is a rare inherited disorder of retinal angiogenesis, leading to incomplete vascularization of the peripheral retinal and poor vascular differentiation. FEVR most often presents in childhood but may progress at any age with sight-threatening manifestations. It is characterized by abnormal retinal vascular development with progressive vitreoretinal features, including retinal capillary dropout, vessel dragging; retinal folds, vessel leakage and exudation; hemorrhage, neovascularization, vitreoretinal interface changes, and serous, tractional, or combined retinal detachment—all contributing to or causing visual impairment and blindness. Currently there are no approved pharmacological therapies for FEVR. 

Noregen is a synthetic targeted growth-factor for retina-related vision loss modeled after norrin, a naturally occurring human protein that guides retinal formation in fetal development. Noregen may promote development of normal, organized blood vessels and neurons in the human eye, ear, and central nervous system for individuals with inherited or acquired retinal diseases and potential applications in other neurosensory diseases.  It is a first-in-class therapeutic candidate to regenerate and repair the retinal vasculature in order to restore and preserve vision in patients with ischemic vitreoretinopathies.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes Noregen eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Bluebird Bio in November sold its priority review voucher to Argenx for $102 million.

“Obtaining U.S. FDA’s Rare Pediatric Disease designation for Noregen in FEVR, after securing Orphan Drug designations, enables us to accelerate our development progress,” said Walter Capone, CEO of Caeregen Therapeutics.  “Regulatory agency endorsement of our urgent mission to help patients with FEVR at risk of vision loss provides critical momentum on our path to the clinic.”

Author: Rare Daily Staff

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