Abstract
In this review period, there were major late-stage development activities with the U.S. Food and Drug Administration (US FDA), including the submission of a New Drug Application (NDA) for a novel treatment for an inherited retinal disease, a pending resubmission of an application previously not approved based upon an appeal, and an ophthalmic drug receiving designation under a new rapid development program for rare diseases. Several other novel therapies are in various stages of clinical development.
Ophthalmic Pharmaceuticals and Biologics
Belite Bio initiated a rolling submission of an NDA to the US FDA for tinlarebant, its oral therapy for the treatment of Stargardt disease type 1. The firm recently presented data from its Phase 3 DRAGON study. The firm also receive orphan drug status for this product in Switzerland (April/May 2026). Cloudbreak Pharma reported that the final patient has completed the 12-month assessment in its global phase 3 trial of CBT-001 (a topical kinase inhibitor) for pterygium (June 2026). Drug Farm announced results from its Phase 1b study of DF-003, an oral ALPK1 inhibitor, for the treatment of retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache syndrome. The firm received designation from the US FDA for its Rare Disease Evidence Principles Process (RDEP, May 2026). Outlook Therapeutics received an appeal from the US FDA with regard to their Biologics License Application (BLA) for Lytenava™ for the treatment of neovascular age-related macular degeneration. The firm will be resubmitting the BLA (May 2026). TearSolutions received US FDA Orphan Drug Designation and Fast Track Designation to its lead investigational therapy, Lacripep™ (lacritin), for the treatment of neurotrophic keratitis. They have begun dosing in a clinical study of this product (June 2026). Therini Bio started dosing in a phase 1b clinical trial of THN391, a fibrin-targeting monoclonal antibody for the treatment of diabetic macular edema (DME, May 2026).
Gene and Cell Therapy
PulseSight reported phase 1 results for PST-611, a gene therapy for transferrin, in patients with geographic atrophy (May 2026).
Regulatory, Government, and Pharmaceutical Industry
Tarsier Pharma is continuing to develop the immunomodulatory compound dazdotuftide for the treatment of noninfectious anterior uveitis (May 2026). The Montana State Department of Public Health and Human Services published proposed rulemaking proposing the adoption of new rules to allow for provision of Montana Senate Bill 535. That bill allowed for licensed experimental treatment centers to offer patients access to experimental treatments that have completed Phase I clinical trials but have not yet received full approval from the US FDA (April 2026). The US FDA:
Issued a guidance on Content of Human Factors Information in Medical Device Marketing Submissions (May 2026). Renewed calls for Amgen’s Tavneos® (avacopan for vasculitis) to be pulled from the market, saying it has discovered new evidence that study personnel doctored the results of the drug’s pivotal study in order to make it look effective (April 2026).
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmic, pharmaceutical, and medical device firms.
Funding Information
No funding was received for this article.