The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for cyclosporine topical ophthalmic emulsion 0.1% (Santen Inc.) for the treatment of severe vernal keratoconjunctivitis in patients aged 4 to 18 years.
Vernal keratoconjunctivitis (VKC) is a rare allergic eye disease that affects the pediatric population and results in severe inflammation of the surface of the eye. The condition is commonly characterized by ocular itching, light sensitivity, redness, and in severe cases, corneal ulcers and vision loss.
The NDA submission includes data from the multicenter, randomized, double-masked, vehicle-controlled phase 3 VEKTIS trial that assessed the efficacy and safety of cyclosporine ophthalmic emulsion in 169 patients aged 4 to 18 years with severe VKC. Patients were randomized to receive either 1 drop of cyclosporine 0.1% 4 times daily (high-dose) or twice daily plus vehicle twice daily (low-dose), or vehicle 4 times daily for the first 4 months. Among the 169 patients enrolled in the trial, 142 entered the 8-month follow-up period receiving either high or low dose cyclosporine. The composite primary end point included corneal fluorescein staining (CFS) score and rescue medication use over 12 months.
Results showed that treatment with high and low dose cyclosporine led to improvements in CFS score, rescue medication use, and quality of life over 4 months. Additionally, patients remained stable on cyclosporine during the 8-month follow-up period, with the high-dose group demonstrating greater benefits in most efficacy measures. As for safety, cyclosporine was found to be well tolerated with instillation site pain being the most common adverse event.
“This is an important milestone for patients with severe vernal keratoconjunctivitis in the US, especially children and adolescents, and the doctors who treat them,” said Peter Sallstig, Senior Vice President and Global Head Product Development Division of Santen. “We look forward to working with the FDA during the review process and are optimistic this treatment could provide much needed symptom relief for those affected so they may better engage in their daily activities.”
A Prescription Drug User Fee Act (PDUFA) target date of June 26, 2021 has been set for this application.
For more information visit santenusa.com.
- Santen announces US FDA filing acceptance of New Drug Application (NDA) for cyclosporine topical ophthalmic emulsion, 0.1% for the treatment of severe vernal keratoconjunctivitis in patients ages 4-18. [press release]. Emeryville, CA: Santen Inc; October 26, 2020.
- Bremond-Gignac D, Doan S, Amrane M, et al. 12-month results of cyclosporine A cationic emulsion in a randomized study in patients with pediatric vernal keratoconjunctivitis. American Journal of Ophthalmology. Published online November 23, 2019. doi: 10.1016/j.ajo.2019.11.020.
This article also appears on Ophthalmology Advisor.