Abstract
Ophthalmical Pharmaceuticals and Biologics
Aldeyra announced results in a new phase 3 trial of its reproxalap for dry eye disease (August 2024).
Annexon began subject dosing in its phase 3 ARCHER II trial evaluating ANX007 for the treatment of geographic atrophy (GA, August 2024).
Avanzanite received marketing authorization from the European Commission for its Akantior™ (polihexanide) for the treatment of acanthamoeba keratitis (August 2024).
Lenz Therapeutics submitted a new drug application to the U.S. FDA for its LNZ100 (aceclidine) for the treatment of presbyopia (August 2024).
Ocular Therapeutix announced agreement with the U.S. FDA on the design of its phase 3 SOL-R clinical trial of its Axpaxli (axitinib intravitreal implant, also known as OTX-TKI) for the treatment of patients with wet AMD, August 2024).
Ocuphire began subject dosing in its VEGA-3 Phase 3 clinical trial evaluating Phentolamine Ophthalmic Solution 0.75% for presbyopia (September 2024).
Sandoz received U.S. FDA approval for its Enzeevu™ (aflibercept-abzv), an Eylea biosimilar, for the treatment of wet age-related macular degeneration (AMD). The approval includes both a 2 mg vial kit and a pre-filled syringe for intravitreal injection (August 2024).
Gene and Cell Therapy
Atsena received rare pediatric disease (RPD) designation for its gene therapy product candidate ATSN-201 for the treatment of X-linked retinoschisis (August 2024).
Bayer subsidiary BlueRock Therapeutics’ investigational new drug application for its OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived therapy designed to treat primary photoreceptor diseases was cleared by the U.S. FDA (September 2024).
Opus Genetics received rare pediatric disease (RPD) designation for its gene therapy product candidate OPGx-LCA5 for the treatment of Leber Congenital Amaurosis Type 5 (August 2024).
Regenxbio licensed rights to two of its gene therapy programs (RGX-381 and RGX-181, now designated as TTX-381 and TTX-181) to Tern Therapeutics. These investigational therapies are designed to deliver a working copy of the TPP1 gene for patients with CLN2 disease, an inherited disorder affecting the central nervous system (August 2024).
Regulatory, Government, and Pharmaceutical Industry
Alcon announced an agreement with China-based Ocumension Therapeutics for commercialization rights in China to a portfolio of Alcon’s dry eye treatments and procedural drops (August 2024).
Formosa Pharmaceuticals announced an agreement with Apotex for commercialization in Canada of its Clobetasol Propionate Ophthalmic Suspension for post-operative use (August 2024).
Ocuvex Therapeutics will be acquiring Visiox Pharmaceuticals, whose lead asset is Omlonti® (omidenepag isopropyl ophthalmic solution 0.002%), an FDA-approved EP2 receptor agonist for ocular hypertension and open-angle glaucoma (August 2024).
U.S. Government:
In execution of the Inflation Reduction Act, the Centers for Medicare and Medicaid Services announced prices for 10 of the top drugs (August 2024). The Federal Trade Commission expressed support for the FDA’s recent draft guidance on interchangeable biosimilar drugs, which proposes changes that could streamline the approval process for biosimilars (August 2024).
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmical, pharmaceutical, and medical device firms. A complete listing of all of Dr. Novack’s consulting affiliations can be found online.
In this review period, two ocular gene therapy products received U.S. FDA rare pediatric disease (RPD) designation, and another cleared FDA review to start clinical trials, and a biosimilar of aflibercept was approved for marketing. A pharmaceutical therapy for acanthamoeba keratitis was approved in Europe, a New Drug Application (NDA) was submitted to the FDA for a cholinergic agonist to treat presbyopia, and several firms began Phase 3 trials.