Rob Volansky
2026-01-23 20:22:00
January 23, 2026
1 min read
Key takeaways:
- Urcosimod 0.05% has been granted compassionate use designation for a patient with neuropathic corneal pain.
- The treatment will be studied in a phase 2b/3 study later this year.
The FDA has authorized urcosimod 0.05% for single-patient expanded access, or compassionate use, in a patient with neuropathic corneal pain, according to a press release from Okyo.
The terms of the Investigational New Drug (IND) application stipulate that urcosimod will be provided by the company to be used in a patient with limited therapeutic options and no FDA-approved treatment options.
“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,” Pedram Hamrah, MD, vice chair of academic medicine in the department of ophthalmology, University of South Florida, said in the release.
Hamrah filed the IND with the FDA for use of the medication in this patient.
“Neuropathic corneal pain is a complex condition involving both immune-mediated inflammation and dysfunctional nerve signaling,” he said. “Urcosimod’s proposed dual mechanism of action, potentially targeting both pathways provides a strong scientific rationale for investigation in this setting.”
“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,” Okyo CEO Robert J. Dempsey said in the release. “This also reflects the potential new hope for patients battling this debilitating painful condition with no FDA-approved treatment available today.”
The FDA granted fast track designation to urcosimod to treat neuropathic corneal pain in May 2025.
A phase 2b/3, multiple dose study involving 120 patients is planned for urcosimod later this year, according to the release.
“Urcosimod is a lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor,” Okyo said. This protein receptor can be found in the immune cells of the eye and may result in an inflammatory response.
A previous randomized, placebo-controlled, double-masked phase 2 study of 18 patients with neuropathic corneal pain demonstrated a statistically significant benefit of urcosimod over placebo in pain reduction. Moreover, the drug also yielded statistically significant improvements in multiple endpoints in another phase 2 study involving 240 patients with dry eye disease, according to the release.
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