FDA Greenlights Urcosimod 0.05% for Patient With Severe Neuropathic Corneal Pain

OKYO Pharma Limited has received FDA authorization for single-patient expanded access to Urcosimod 0.05%, a groundbreaking development for treating one patient suffering from severe neuropathic corneal pain (NCP). The request was championed by Dr. Pedram Hamrah at the University of South Florida, representing a critical step forward in addressing an unmet medical need that affects countless patients with no viable therapeutic options.

A Rare Neuropathic Condition Leaves Patients Without Effective Solutions

Neuropathic corneal pain represents one of the most challenging conditions in ophthalmology—a chronic affliction characterized by nerve dysfunction and corneal inflammation that can be profoundly debilitating. What makes this condition particularly tragic is the near-total absence of FDA-approved treatments specifically designed to combat it. Currently, patients are forced to rely on off-label medications that frequently deliver disappointing results, leaving them in a state of persistent suffering with minimal relief.

The case approved by the FDA involves a patient whose condition has become so severe that conventional alternatives have been exhausted. Under the compassionate-use program, Urcosimod 0.05% will now be available to provide hope where traditional therapies have failed.

Urcosimod’s Dual Mechanism Addresses Both Inflammation and Pain Signaling

What distinguishes Urcosimod 0.05% is its innovative dual-action approach to tackling neuropathic corneal pain. Unlike single-target treatments, this formulation simultaneously targets inflammatory pathways while addressing nerve-related pain signaling—a comprehensive strategy that preclinical studies have demonstrated to be highly effective. The drug’s anti-inflammatory and pain-reducing properties have shown encouraging activity in laboratory models, supporting its potential efficacy in real-world clinical applications.

The 0.05% concentration represents years of pharmaceutical optimization, carefully calibrated to balance therapeutic potency with tolerability in ocular applications. Early Phase 2 trial results in NCP patients further validated this approach, demonstrating that Urcosimod holds genuine promise for transforming treatment outcomes.

Accelerating Toward Broader Patient Access

Building on this momentum, OKYO Pharma has announced plans to launch a 120-patient Phase 2b/3 multiple-dose study later in 2026. This expanded trial represents a critical milestone in moving Urcosimod 0.05% from compassionate use into mainstream clinical evaluation, potentially paving the way for broader FDA approval and patient access.

The authorization of compassionate use serves as validation not only of OKYO’s scientific approach but also of the urgent need for effective NCP therapies. As the company advances its clinical program, the pharmaceutical and ophthalmology communities are closely watching developments that could finally bring relief to a patient population that has long been overlooked by therapeutic innovation.

Investors tracking OKYO have noted significant stock volatility, with the company’s shares experiencing notable movement in response to clinical progress announcements, underscoring the market’s recognition of the potential importance of Urcosimod 0.05% in addressing this underserved treatment space.