Glaukos Receives FDA Acceptance for Epioxa™ NDA Submission
Glaukos Corporation (NYSE: GKOS), a leading ophthalmic pharmaceutical and medical technology company specializing in innovative therapies for the treatment of glaucoma, corneal disorders, and retinal diseases, has announced a significant regulatory milestone. The company has received the “Day 74” notification from the U.S. Food and Drug Administration (FDA), confirming that its recently submitted New Drug Application (NDA) for Epioxa™ (Epi-on) is sufficiently complete to allow a substantive review. Epioxa is an advanced, next-generation corneal cross-linking iLink therapy developed for the treatment of keratoconus, a sight-threatening corneal disease. The FDA has set the Prescription Drug User Fee Act (PDUFA) target date for completing the review of the Epioxa NDA as October 20, 2025, following a standard 10-month review period, aligning with Glaukos’ expectations.
The acceptance of the NDA submission marks a major milestone for Glaukos and the broader ophthalmic community. Thomas Burns, Glaukos’ Chairman and Chief Executive Officer, expressed optimism regarding this regulatory progress. “The acceptance of the Epioxa NDA represents another important step in being able to provide keratoconus patients and the ophthalmic community with the first FDA-approved, non-invasive corneal cross-linking drug therapy that does not require removal of the corneal epithelium, the outermost layer of the front of the eye,” Burns stated. He emphasized the potential benefits of Epioxa, including reduced procedure times, improved patient comfort, and shorter recovery periods, all of which could significantly enhance the standard of care for keratoconus patients.
Epioxa’s NDA submission is backed by robust clinical data derived from two pivotal Phase 3 trials. These trials successfully met their pre-specified primary efficacy endpoints, demonstrating that the therapy effectively strengthens corneal tissue and halts disease progression while maintaining a favorable tolerability and safety profile. The compelling clinical evidence supports Glaukos’ confidence in Epioxa as a transformative therapeutic option for keratoconus patients.
Keratoconus is a progressive and debilitating eye condition that primarily affects adolescents and young adults. It is characterized by the thinning and weakening of the cornea, leading to visual distortion, increased sensitivity to light, and, in severe cases, significant visual impairment or blindness. If left untreated, keratoconus is one of the leading causes of corneal transplant (penetrating keratoplasty) in the United States. Statistics indicate that approximately 90% of keratoconus cases affect both eyes, and as many as 20% of patients eventually require corneal transplants. The impact of the disease underscores the need for effective treatment options that go beyond traditional symptomatic relief, such as eyeglasses and contact lenses.
Glaukos has been at the forefront of innovation in corneal cross-linking therapies. The company’s first-generation iLink therapy, marketed as Photrexa® (Epi-off), is the first and only FDA-approved treatment shown to slow or halt keratoconus progression. The iLink therapy is supported by over 300 peer-reviewed publications validating its performance and safety. However, the Epi-off procedure requires the removal of the corneal epithelium, which can lead to prolonged recovery times and discomfort for patients. Epioxa, Glaukos’ next-generation therapy, is designed to address these limitations by allowing corneal cross-linking without epithelial removal.
Epioxa utilizes a proprietary, novel drug formulation engineered to penetrate the epithelial layer of the cornea without requiring its removal. Additionally, the therapy employs a stronger UV-A irradiation protocol and supplemental oxygen to enhance the cross-linking process, thereby fortifying corneal tissue more efficiently. If approved, Epioxa would become the first FDA-approved, non-invasive corneal cross-linking therapy, marking a paradigm shift in the treatment landscape for keratoconus. By eliminating the need for epithelial removal, the therapy is expected to significantly improve the patient experience by minimizing procedure-related discomfort, reducing recovery times, and streamlining the treatment process.
The anticipated approval of Epioxa aligns with Glaukos’ broader mission to revolutionize ophthalmic care through groundbreaking medical technologies. Over the years, the company has developed a strong reputation for pioneering innovative therapies, particularly in the realm of micro-invasive glaucoma surgery (MIGS), corneal health, and retinal disease management. Epioxa represents yet another testament to Glaukos’ commitment to addressing unmet medical needs and enhancing patient outcomes.
Beyond clinical benefits, the potential approval of Epioxa carries significant commercial implications for Glaukos. The global keratoconus treatment market is expanding, driven by increasing disease prevalence, greater awareness among patients and healthcare professionals, and advancements in treatment modalities. According to industry analysts, the market for corneal cross-linking therapies is poised for substantial growth, and Epioxa’s non-invasive approach could position it as a preferred choice among ophthalmologists and patients alike.
The regulatory journey for Epioxa now moves into the next critical phase, with the FDA conducting a comprehensive review of the submitted data. Throughout this process, Glaukos plans to work closely with the agency, ensuring that all regulatory requirements are met to facilitate a smooth approval pathway. While the PDUFA goal date is set for October 20, 2025, the company remains optimistic about the outcome and is preparing for a potential commercial launch upon approval.
The ophthalmic community and patients suffering from keratoconus will be watching this regulatory development closely, as the approval of Epioxa could redefine the treatment paradigm for this sight-threatening disease. By offering a non-invasive, patient-friendly alternative to existing corneal cross-linking therapies, Epioxa has the potential to improve the quality of life for thousands of keratoconus patients, reducing the need for more invasive interventions such as corneal transplants.
In conclusion, the FDA’s acceptance of Glaukos’ NDA submission for Epioxa marks a pivotal step toward advancing keratoconus treatment. With a strong foundation of clinical data, a novel non-invasive approach, and a commitment to improving patient outcomes, Glaukos is well-positioned to lead the next generation of corneal cross-linking therapies. The ophthalmic industry eagerly awaits the FDA’s decision, which could pave the way for a new era in keratoconus management.
