Glaukos Corporation announced that the U.S. Food and Drug Administration has approved its New Drug Application for Epioxa HD/Epioxa, marking a significant breakthrough in keratoconus treatment. This approval establishes Epioxa as the first and only FDA-approved, incision-free, topical drug therapy for keratoconus that does not require removal of the corneal epithelium.
The novel therapy represents a transformative innovation in corneal cross-linking, offering an alternative to traditional procedures that have historically required epithelial removal. Epioxa is based on two oxygen-enriched topical formulations—Epioxa HD and Epioxa—that are sequentially administered during the cross-linking procedure and activated by UV light in an oxygen-enriched environment.
Clinical Trial Results Support Approval
The FDA approval is supported by robust clinical evidence from two prospective, randomized, multicenter, double-masked Phase 3 pivotal trials that enrolled more than 400 patients. Both trials successfully achieved their pre-specified primary efficacy endpoints and demonstrated favorable tolerability and safety profiles.
"The FDA approval of Epioxa ushers in a new standard-of-care for patients suffering from keratoconus with the first FDA-approved topical drug therapy that does not require removal of the corneal epithelium," said Thomas Burns, Glaukos chairman and chief executive officer. "Epioxa is designed to significantly improve patient comfort and minimize recovery time, representing a game-changing new treatment for patients suffering from keratoconus."
Addressing Critical Unmet Medical Need
Keratoconus is a debilitating eye condition characterized by progressive thinning and weakening of the cornea that is often most aggressively advancing in patients under the age of 30. If left untreated, keratoconus can lead to loss of visual function and even blindness and is one of the leading causes of corneal transplants in the United States. Approximately 90% of cases are bilateral and as many as 20% of untreated patients ultimately require a corneal transplant.
"Keratoconus is currently an underdiagnosed and undertreated disease. For keratoconus patients who are fortunate enough to be diagnosed, the current standard-of-care requires removal of the epithelium, the top layer of the cornea. The pain and extended healing time associated with the current surgical procedure are major barriers to adoption," said W. Barry Lee, MD, corneal specialist at Eye Consultants of Atlanta and President of the Cornea Society. "As an incision-free treatment that does not require removal of the epithelium, I expect the newly approved Epioxa treatment to address both of these major concerns."
Clinical Expert Perspective
Rajesh K. Rajpal, MD, founder of See Clearly Vision Group in McLean, VA, described the therapy as "a transformative product that sets a new standard for keratoconus treatment." He noted that "it's the first and only FDA-approved incision-free procedure using bioactive topical drops catalyzed by supplemental oxygen and UV light, designed to slow or halt disease progression of keratoconus in a single administration."
Rajpal emphasized that the therapy "is more efficient, eliminates the pain associated with epithelium removal, and is associated with better quality of life, while also reducing the downstream medical costs associated with disease progression and deterioration of eyesight."
Safety Profile and Administration
The most common adverse reaction observed in clinical trials was conjunctival hyperemia, occurring in 31% of patients. Other adverse reactions occurring in 5% to 25% of eyes included corneal opacity (haze), photophobia, punctate keratitis, eye pain, eye irritation, increased lacrimation, corneal epithelium defect, eyelid edema, corneal striae, visual acuity reduced, dry eye, and anterior chamber flare.
Epioxa HD and Epioxa are indicated for use in epithelium-on corneal collagen cross-linking for the treatment of keratoconus in adults and pediatric patients aged 13 years and older, in conjunction with the O2n System and the Boost Goggles.
Commercial Launch and Access Initiatives
Glaukos expects Epioxa to be commercially available in the first quarter of 2026. The company plans to make substantial investments in patient awareness and access while addressing the longstanding challenges of underdiagnosis and undertreatment that have affected this rare disease patient community.
Key initiatives include streamlined patient access support programs, a co-pay assistance program to reduce financial barriers, integrated healthcare professional and patient-centric strategies to improve education and engagement, and broad awareness and detection programs aimed at earlier and more widespread screening for and diagnosis of the disease.
This approval builds upon Glaukos' existing corneal cross-linking portfolio. The company's first-generation therapy, Photrexa Viscous/Photrexa, which requires removal of the corneal epithelium, received FDA approval in 2016 as an orphan drug and has been supported by more than 300 peer-reviewed publications.
