FDA Approves Epioxa for treatment of Keratoconus

“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. We appreciate the clinical investigators and study participants in the clinical trials for their instrumental roles in helping us reach this important advancement. This approval marks a major milestone in our mission to improve patient access to sight-saving therapies, and we are excited to bring this transformative therapy to market for the benefit of patients.”

“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.”

The FDA approval of Epioxa is based on results from two prospective, randomized, multicenter, double-masked, Phase 3 pivotal trials that randomized a total of over 400 patients. Both trials successfully achieved their pre-specified primary efficacy endpoints and demonstrated favorable tolerability and safety profiles.

Glaukos intends for Epioxa to be commercially available in the first quarter of 2026. With this approval, Glaukos 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. These efforts will be focused on supporting patients and families across every step of the journey, from awareness to diagnosis and treatment. Key initiatives include streamlined patient access support programs, a co-pay assistance program to reduce financial barriers, integrated healthcare professional (HCP) 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.

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 (penetrating keratoplasty) in the United States. Approximately 90% of cases of keratoconus are bilateral and as many as 20% of untreated keratoconus patients ultimately require a corneal transplant. Conventional keratoconus treatments such as eyeglasses or contact lenses address visual symptoms only and do not slow or halt underlying disease progression.

Glaukos’ first-generation corneal cross-linking therapy, known as Photrexa® Viscous / Photrexa®, which requires removal of the corneal epithelium, received U.S. FDA approval in 2016 as an orphan drug and has since been the first-and-only FDA-approved corneal cross-linking therapy for the treatment of keratoconus. There are more than 300 peer-reviewed publications supporting the performance and safety of Glaukos’ cross-linking therapies.