FDA Clears OKYO's Urcosimod for Compassionate Use in Severe Neuropathic Corneal Pain

OKYO Pharma Limited received FDA authorization for single-patient expanded access to Urcosimod 0.05%, marking a significant step toward addressing severe neuropathic corneal pain. The groundbreaking approval allows a patient with debilitating symptoms and no viable conventional treatment options to receive the investigational therapy under emergency patient access protocols. This compassionate-use authorization represents an important development in pain management for a condition where current therapeutic options remain severely limited.

Understanding Neuropathic Corneal Pain and Current Treatment Gaps

Neuropathic corneal pain is a chronic and often severe condition characterized by nerve dysfunction and corneal inflammation, resulting in persistent discomfort that significantly impacts patient quality of life. The condition presents a substantial clinical challenge because currently no FDA-approved medications specifically target this type of pain. Patients suffering from neuropathic corneal pain have historically relied on off-label drug treatments that frequently provide inadequate relief, leaving a critical gap in therapeutic options. This unmet medical need was the driving force behind the FDA’s decision to authorize expanded access for Urcosimod, as submitted by Pedram Hamrah, MD, at the University of South Florida.

Urcosimod’s Dual Mechanism Against Corneal Pain Signals

Urcosimod, previously designated as OK-101, employs a distinctive dual-targeted approach that addresses both the inflammatory component and nerve-related pain signaling pathways involved in neuropathic corneal pain. Preclinical laboratory studies demonstrated that the compound possessed notable anti-inflammatory and pain-reducing properties. The mechanism’s dual action makes it particularly promising for complex pain conditions like neuropathic corneal pain, where inflammation and neural dysfunction intertwine. Most significantly, Urcosimod recently demonstrated clinical efficacy in a randomized Phase 2 trial enrolling neuropathic corneal pain patients, providing evidence that the dual mechanism translates to real therapeutic benefit in actual patient populations.

Advancing Toward Broader Patient Access

Building on positive clinical trial data, OKYO Pharma plans to launch a larger 120-patient Phase 2b/3 multiple-dose study later in 2026, aimed at further evaluating the drug’s effectiveness and safety profile. This expanded clinical program represents the next phase in establishing whether Urcosimod can become the first FDA-approved therapy specifically designed for neuropathic corneal pain management. The combination of compassionate-use approval and planned Phase 2b/3 expansion underscores the medical community’s recognition that neuropathic corneal pain requires dedicated therapeutic solutions, and OKYO’s investigational approach offers renewed hope for patients currently left without adequate pain relief options.