FDA Authorizes Urcosimod for Neuropathic Corneal Pain – Compassionate Use

On January 23, 2026, the FDA authorized expanded access, often referred to as compassionate use, to the investigational treatment urcosimod 0.05% for a single patient suffering from neuropathic corneal pain. This decision allows a physician to utilize the drug in a case where existing treatment options have proven insufficient.

Understanding the Authorization

The authorization stems from an Investigational New Drug (IND) application, meaning Okyo, the developer of urcosimod, will provide the medication for use in a patient facing limited therapeutic alternatives and lacking access to any FDA-approved treatments for their condition. This pathway is reserved for serious or life-threatening conditions when standard treatments are unavailable.

Did You Know? Urcosimod is a lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor, a protein receptor found in the immune cells of the eye.

The Potential of Urcosimod

Pedram Hamrah, MD, vice chair of academic medicine in the department of ophthalmology at the University of South Florida, filed the IND application and expressed gratitude for the FDA’s authorization. Dr. Hamrah believes exploring urcosimod’s potential is crucial, given the complex nature of neuropathic corneal pain, which involves both inflammation and nerve signaling dysfunction.

According to Okyo CEO Robert J. Dempsey, this compassionate use authorization underscores the urgent need for innovative therapies specifically designed to address neuropathic corneal pain, offering potential hope to patients currently battling this debilitating condition.

Previous Research and Future Plans

Urcosimod previously received fast track designation from the FDA in May 2025 for the treatment of neuropathic corneal pain. Phase 2 studies have shown promising results: a randomized, placebo-controlled, double-masked phase 2 study of 18 patients demonstrated a statistically significant benefit in pain reduction. Additionally, a separate phase 2 study involving 240 patients with dry eye disease also yielded statistically significant improvements with urcosimod.

Looking ahead, Okyo plans to initiate a phase 2b/3 study involving 120 patients later this year to further evaluate the efficacy and safety of urcosimod.

Expert Insight: The FDA’s compassionate use authorization highlights the critical need for new treatments for conditions like neuropathic corneal pain, where existing options are limited. This pathway allows for potential benefit to a patient while simultaneously gathering valuable data that could inform future clinical trials and regulatory decisions.

Frequently Asked Questions

What is compassionate use?

Compassionate use, or expanded access, allows patients with serious or life-threatening conditions to access investigational medical products when no comparable or satisfactory alternative treatment options are available.

What is neuropathic corneal pain?

Neuropathic corneal pain is a complex condition involving both immune-mediated inflammation and dysfunctional nerve signaling, causing significant pain and discomfort.

What are the next steps for urcosimod?

Okyo plans to begin a phase 2b/3 study involving 120 patients later this year to further investigate the potential of urcosimod for treating neuropathic corneal pain.

How might advancements in treatments for rare conditions like neuropathic corneal pain impact the broader landscape of personalized medicine?