FDA Greenlights Novel Eye Pain Drug for Compassionate Use

LONDON and NEW YORK – January 23, 2026 – In a significant move that underscores a pressing unmet medical need, the U.S. Food and Drug Administration (FDA) has authorized the compassionate use of an investigational drug for a patient suffering from severe neuropathic corneal pain (NCP). The decision allows a single patient with limited therapeutic options to be treated with urcosimod, a novel therapy being developed by the clinical-stage biopharmaceutical company OKYO Pharma.

The authorization was granted through a single-patient expanded access Investigational New Drug (IND) application submitted by Dr. Pedram Hamrah, a leading expert in the field, now at the University of South Florida. This regulatory step provides a potential lifeline for an individual battling a debilitating condition for which there are currently no FDA-approved treatments, while also casting a spotlight on a promising new therapeutic approach.

The Agonizing Reality of Neuropathic Corneal Pain

Neuropathic corneal pain is a chronic and often incapacitating condition that transforms the simple act of opening one's eyes into an ordeal. Patients frequently describe the sensation not as mere discomfort, but as severe, unrelenting pain—a feeling of burning, stabbing, or having a foreign object, like a knife, lodged in the eye. This pain can be accompanied by extreme sensitivity to light (photophobia) and may radiate to the face and head, severely diminishing a person's quality of life.

The condition is believed to stem from damage or dysfunction of the sensory nerves in the cornea, the eye's clear outer layer. This nerve damage is often coupled with inflammatory processes, creating a complex and difficult-to-treat pathology. Because its symptoms can mimic those of severe dry eye disease, NCP is often misdiagnosed, leading to prolonged suffering and ineffective treatment cycles.

For the thousands affected, the therapeutic landscape is bleak. With no FDA-approved drugs specifically for NCP, clinicians and patients are forced to navigate a maze of off-label options. These can include everything from specialized tear formulations and topical corticosteroids to systemic neurological drugs like gabapentin and antidepressants. Unfortunately, for many, these treatments provide little to no relief, leaving them in a state of constant pain with few places to turn.

A Lifeline Through Compassionate Use

The FDA's Expanded Access program, commonly known as compassionate use, exists for these exact scenarios. It provides a pathway for patients with serious or life-threatening conditions to access investigational medical products outside of clinical trials when no comparable or satisfactory alternative therapies are available. The decision to grant access requires that the potential benefit to the patient justifies the potential risks of an unapproved therapy, and both the treating physician and the drug's manufacturer must be in agreement.

This authorization was championed by Dr. Pedram Hamrah, a renowned ophthalmologist who serves as Vice Chair of Academic Medicine at the University of South Florida's Department of Ophthalmology. His physician-sponsored IND application highlights a direct and urgent clinical need identified by a specialist on the front lines of patient care.

“We are grateful that this FDA authorization allows us to explore the potential of urcosimod in a patient with severe neuropathic corneal pain through a physician-sponsored expanded access IND,” said Dr. Hamrah. “Neuropathic corneal pain is a complex condition involving both immune-mediated inflammation and dysfunctional nerve signaling. Urcosimod’s proposed dual mechanism of action, potentially targeting both pathways provides a strong scientific rationale for investigation in this setting.”

Urcosimod: A Novel Approach to Pain and Inflammation

Urcosimod (formerly known as OK-101) represents a new frontier in the treatment of ocular pain and inflammation. It is a lipid-conjugated chemerin peptide agonist, a complex name for a molecule designed to perform a highly specific, dual-action role. The drug targets the ChemR23 receptor, which is found on both the immune cells that drive inflammation and the nerve cells responsible for transmitting pain signals.

By engaging this receptor, urcosimod is believed to simultaneously quell the inflammatory response and modulate the dysfunctional nerve signaling that causes the perception of pain. This dual mechanism is particularly well-suited for a condition like NCP, where both inflammation and nerve damage are key drivers of the disease.

The scientific rationale is supported by encouraging clinical data. OKYO Pharma recently announced positive top-line results from a randomized, placebo-controlled Phase 2 trial in 18 NCP patients, where Dr. Hamrah served as the principal investigator. In that study, 75% of patients treated with urcosimod showed a reduction in pain scores of over 80% after 12 weeks. Perhaps more remarkably, the study also revealed evidence of corneal nerve restoration in the urcosimod group—an increase in nerve fiber count and length that was not observed in patients receiving the placebo. This suggests the drug may not only manage symptoms but also address the underlying nerve damage.

A Strategic Step on the Path to Approval

While this compassionate use authorization directly benefits only a single individual, its implications are far-reaching. It serves as a powerful signal of the urgent need for new NCP therapies and provides a vote of confidence in urcosimod's potential. The drug has already been granted Fast Track designation by the FDA, a program designed to facilitate the development and expedite the review of drugs intended to treat serious conditions and fill an unmet medical need.

“Supporting physician-sponsored expanded access to urcosimod under compassionate use in the U.S. highlights the pressing global need for innovative therapies to specifically address neuropathic corneal pain,” said Robert J. Dempsey, Chief Executive Officer of OKYO Pharma. “This also reflects the potential new hope for patients battling this debilitating painful condition with no FDA approved treatment available today.”

Building on its positive Phase 2 results, OKYO Pharma is preparing to advance urcosimod into a larger, 120-patient Phase 2b/3 study later this year. This pivotal trial is designed to gather the robust data necessary to define a clear path toward potential regulatory approval. The real-world experience gained from this single-patient compassionate use case, while not formal trial data, contributes valuable insights and reinforces the drug's therapeutic promise as it moves into the next critical stage of its clinical journey.