Abstract
In this review period, an allogenic encapsulated cell-based intravitreal implant containing retinal pigment epithelial cells (ARPE) expressing recombinant human ciliary neurotrophic factor was approved by the U.S. FDA for the treatment of idiopathic macular telangiectasia type 2. The basic science work on ARPE cells was conducted at UC Davis nearly 30 years ago, 1 and the product-entered ophthalmical clinical trials were conducted 20 years ago. 2 The pivotal efficacy studies used a structural retinal endpoint, the width of the ellipsoid zone. In other news items, a low concentration of atropine was approved for slowing myopic progression; results from another formulation of atropine for the same indication were announced and are now under review by the U.S. FDA for approval, as were results from a novel dry eye molecule.
Ophthalmical Pharmaceuticals and Biologics
Astellas received U.S. FDA expanded approval for its Izervay® (avacincaptad pegol intravitreal solution), for the treatment of geographic atrophy, allowing the drug to be used without a limitation on duration of dosing (February 2025). Glaukos announced that the U.S. FDA has accepted for review its New Drug Application (NDA) for Epioxa™ (Epi-on), its corneal cross-linking therapy for the treatment of keratoconus (February 2025). IVIEW Therapeutics announced results from its Phase 1/2 clinical trial of IVW-1001, a TRPM8 agonist, for the treatment of dry eye disease (February 2025). Opthea completed patient visits in its COAST Phase 3 trial of sozinibercept in combination with aflibercept for the treatment of wet age-related macular degeneration (AMD) (February 2025). Opus Genetics (formerly Ocuphire) completed enrollment is its VEGA-3 Phase 3 clinical trial evaluating phentolamine ophthalmic solution 0.75% for presbyopia. The firm is also enrolling subjects in its trial of this product for the treatment of visual loss in low light conditions associated with keratorefractive surgery. Finally, the firm was granted a Fast Track designation for this product for this indication of treatment of significant chronic night driving impairment (February 2025). Santen received approval in Japan for a low concentration of atropine for slowing myopic progression (February 2025). Sydnexis announced results of a trial of its low concentration of atropine for slowing myopia progression (February 2025) and acceptance for review of an NDA for this product (March 2025).
Gene and Cell Therapy
Adverum started the ARTEMIS Phase 3 study designed to evaluate the efficacy and safety of Ixo-vec (ixoberogene soroparvovec), a gene therapy delivered by intravitreal injection for the treatment of neovascular (wet) AMD (March 2025). Neurotech received U.S. FDA approval for revakinagene taroretcel-lwey, an allogenic encapsulated ARPE cell-based intravitreal implant (Encelto™) for the treatment of idiopathic macular telangiectasia type 2 (March 2025).* (Huang et al.
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Regulatory, Government, and Pharmaceutical Industry
The U.S. Centers for Disease Control instructed its scientists to retract or pause the publication of any research manuscript being considered by any medical or scientific journal (February 2025). The U.S. National Institutes of Health announced a simplified framework for NIH peer review criteria (March 2025).
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmical, pharmaceutical, and medical device firms. A complete listing of all of G.D.N.’s consulting affiliations can be found online.
Funding Information
No funding was received for this article.
*Indicates news item of relevance to article in current issue.