27 Jan Caeregen Therapeutics Announces Orphan Drug Designation Granted To Noregen™
A Novel Regenerative Therapy For The Treatment Of Familial Exudative Vitreoretinopathy (FEVR)
- First potential FEVR therapy to obtain U.S. Food and Drug Administration Orphan Drug Designation
- Follows Orphan Medicinal Product Designation by European Commission based European Medicines Agency Committee for Orphan Medicinal Products Positive Opinion
January 27, 2022, Rochester, MI – Caeregen Therapeutics, LLC, a Michigan-based, regenerative medicine therapeutics company, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for Noregen™ (CTR-107), the first potential regenerative therapy for the treatment of Familial Exudative Vitreoretinopathy (FEVR) associated vision loss. This follows the European Commission granting ODD for Noregen™ in FEVR in the European Union (EU) based on a positive opinion issued by the European Medicines Agency Committee for Orphan Medicinal Products (COMP) in late 2021.
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.
“Orphan drug designation from both FDA and EMA is a milestone in the development of Noregen™, highlighting the need for new treatment options for patients with FEVR,” said Michael T. Trese, M.D., co-Founder and Chief Technology Officer of Caeregen Therapeutics. “As a novel regenerative medicine, Noregen™ has the potential to be the first in class treatment to restore the functional pathways activating growth of normal retinal blood vessels and neuronal cells, supporting healthy retinal function” added Dr. Trese.
ODD in the US and EU are intended to encourage the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. US FDA ODD is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain US FDA fees and seven years of post-approval marketing exclusivity. ODD in the EU provides similar benefits and extends marketing exclusivity to ten years.
“We are deeply gratified to obtain orphan drug designation for Noregen™ in FEVR from both the FDA and EMA in such close succession.”,” said Kimberley Drenser, M.D., Ph.D., Chief Scientific Officer of Caeregen Therapeutics. “We believe this designation highlights the potential of Noregen to improve clinical outcomes for patients with FEVR at risk of progressive vision loss.”
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 www.caeregen.com.
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. 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, Noregen™ is currently being developed for Familial Exudative Vitreoretinopathy (FEVR), a rare disease of urgent, unmet medical need. For more information, please visit www.caeregen.com.
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 www.caeregen.com.
Caeregen Therapeutics, LLC.
Walter M. Capone
CEO & President