The United States Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for KPI-121 0.25%, an investigational treatment developed by Kala Pharmaceuticals for dry eye disease. This acceptance initiates a comprehensive review process, with a target action date set for August 15, 2019, under the Prescription Drug User Fee Act (PDUFA). If approved, KPI-121 0.25% would become the first FDA-approved therapy specifically indicated for the temporary relief of both the signs and symptoms of dry eye disease, including acute flare-ups.

A potential breakthrough in dry eye therapy

Kala Pharmaceuticals aims to address a significant unmet need in ophthalmology with KPI-121 0.25%. Unlike existing chronic therapies, this treatment is designed for short-term use, offering a two-week course of therapy to alleviate episodic exacerbations of dry eye symptoms. The company’s proprietary AMPPLIFY Drug Delivery Technology enhances the penetration of the active ingredient, loteprednol etabonate, into ocular tissues, potentially increasing efficacy and patient comfort.

Clinical trials and efficacy

The NDA submission is supported by robust clinical data, including results from one Phase 2 and two Phase 3 trials—STRIDE 1 and STRIDE 2—that collectively involved over 2,000 patients with dry eye disease. These studies evaluated the safety and efficacy of KPI-121 0.25% in providing temporary relief from dry eye symptoms. Additionally, at the FDA’s recommendation, Kala Pharmaceuticals initiated a third Phase 3 trial, STRIDE 3, in July 2018, with topline results expected in the fourth quarter of 2019.

Expert insights on dry eye treatment

Dr. Edward Holland, Director of Cornea Services at the Cincinnati Eye Institute and Professor of Clinical Ophthalmology at the University of Cincinnati, emphasized the importance of this development. He noted that current FDA-approved treatments for dry eye disease are primarily chronic therapies, typically prescribed for patients with persistent symptoms. Dr. Holland highlighted that the majority of patients experience episodic flare-ups characterized by acute exacerbations of signs and symptoms. Therefore, an FDA-approved, safe, and effective short-term treatment like KPI-121 0.25% would represent a significant advancement in patient care.

The AMPPLIFY Drug Delivery Technology

Kala Pharmaceuticals’ AMPPLIFY Drug Delivery Technology is a key innovation in KPI-121 0.25%. This technology utilizes mucus-penetrating particles to enhance the delivery of loteprednol etabonate into target ocular tissues. Preclinical studies have demonstrated that this approach increases drug penetration through the tear film mucins, potentially leading to more effective treatment outcomes for patients suffering from dry eye disease.

Implications for the ophthalmology market

The acceptance of the NDA for KPI-121 0.25% by the FDA marks a pivotal moment in the field of ophthalmology. If approved, this treatment could fill a critical gap in the management of dry eye disease, offering patients a targeted, short-term therapy for acute symptom relief. This development underscores the ongoing innovation within the pharmaceutical industry to address complex ocular conditions and improve patient quality of life.

  AMPPLIFY Drug Delivery Technology, Cincinnati Eye Institute, Edward Holland, FDA, Kala Pharmaceuticals, KPI-121 0.25%, University of Cincinnati