Caeregen Therapeutics Announces FDA Rare Pediatric Disease Designation for Noregen™ in the Treatment Of Familial Exudative Vitreoretinopathy (FEVR)

08 Dec 2022
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First potential FEVR therapy to obtain U.S. FDA Rare Pediatric Disease Designation Designation provides Noregen™ Rare Pediatric Disease Priority Review Voucher Eligibility Follows earlier Orphan Drug Designations in FEVR by both US FDA and EC EMA December 8, 2022, Rochester, MI – Caeregen Therapeutics, LLC, a Michigan-based, regenerative medicine therapeutics company, today announced the U.S. Food and Drug Administration (FDA) designated Noregen™ for treatment of Familial Exudative Vitreoretinopathy (FEVR) as a drug for “rare pediatric disease*”. Through attainment of FDA’s Rare Pediatric Disease (RPD) designation, Noregen™ (CTR-107) is eligible to obtain a Priority Review Voucher at the time of marketing approval. This PRD designation for Noregen™ is the first to be granted by FDA for children with FEVR. The RPD designation for Noregen™ in FEVR follows the attainment of Orphan Drug Designation (ODD) by both the FDA and European Commission (EC) European Medicines Agency (EMA) earlier this year. Noregen™ is a synthetic targeted growth-factor that mimics the properties of norrin, a naturally occurring human protein that promotes development of normal, organized blood vessels and neurons in the human eye, ear, and central nervous system. Noregen™ is being developed for FEVR, a genetic disorder of retinal blood vessel formation, resulting in partial or complete vision loss. When injected into the eye, Noregen™ may regenerate retinal blood vessels and neurons restoring normal retinal function and counteracting vision loss due to FEVR. "The U.S. FDA’s Rare Pediatric Disease Designation for Noregen™ in FEVR provides important validation of our development efforts and the urgent need for new treatment options for patients,” said Kimberly A. Drenser M.D. Ph.D., co-Founder and Chief Scientific Officer of Caeregen Therapeutics. “As new, regenerative medicine activating growth of normal retinal blood vessels and neuronal cells, Noregen™ has the exciting potential to restore, protect and maintain healthy retinal function” added Dr. Drenser. The FDA’s RPD designation is intended to stimulate new drug development for rare pediatric diseases by offering additional incentives for obtaining FDA approval of these products, including eligibility for obtaining a Priority Review Voucher (PRV) that can be used for a subsequent human drug application. Drug companies receiving a PRV can obtain a Priority Review of any drug in their pipeline, which allows for a shorter clock for review of a marketing application (6 months compared with the 10-month standard review), or they can monetize the PRV by selling it to another drug company. Under the current statutory sunset provisions, FDA states that after September 30, 2024, it may only award vouchers for approved rare pediatric disease product applications if companies have an RPD designation for a drug granted by September 30, 2024 and may not award any RPD PRV after September 30, 2026. “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, Chief Executive Officer 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.” ***** *“Rare Pediatric Disease” as defined in section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 360ff(a)(3)) About FAMILIAL EXUDATIVE VITREORETINOPATHY (FEVR) Familial Exudative Vitreoretinopathy, or ‘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 (Ranchod et al., 2011). 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 (Wood et al., 2019) - all contributing to or causing visual impairment and blindness. Currently there are no approved pharmacological therapies for FEVR. For more information, please visit: . About Noregen™ NoregenÔ 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. NoregenÔ 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. As 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, NoregenÔ is currently being developed for Familial Exudative Vitreoretinopathy (FEVR), a rare disease of urgent, unmet medical need. For more information, please visit: . About Caeregen Therapeutics Caeregen Therapeutics, LLC, based in Rochester, MI with offices in Chapel Hill, N.C., is a regenerative medicines company developing therapeutics for neurosensory diseases. By exploiting biological pathways and signaling related to cellular and organ development, Caeregen is focused on advancing targeted therapies with the ability to repair, restore and protect neurosensory tissues affected by inherited or acquired diseases. Caeregen is currently developing Noregen, a unique, novel, recombinant protein mimetic of human norrin-derived growth factor for the potential treatment of retinal-related vision loss. For more information, please visit: . Contacts: Caeregen Therapeutics, LLC. Walter M. Capone CEO & President Email: wmcapone@caeregen.com Mobile: 203-278-1172
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