OKYO Pharma Gets FDA Authorization for Compassionate Use of Urcosimod in Neuropathic Corneal Pain

OKYO Pharma Ltd (NASDAQ: OKYO) has received a major regulatory green light in the United States: the US Food and Drug Administration (FDA) has authorized a single-patient expanded access—often called compassionate use—for the company’s investigational eye therapy, urcosimod, to treat neuropathic corneal pain. The authorization allows a 0.05% dose of urcosimod to be provided to a patient living with severe neuropathic corneal pain who has limited treatment options, especially because there are currently no FDA-approved therapies specifically for this condition.

This development is more than just a headline. It’s a practical step that may help a patient right now, while also adding momentum to OKYO Pharma’s broader clinical program. In the same update, the company emphasized that it will supply urcosimod under the FDA-authorized expanded access program. The company has also previously received FDA authorization for an investigational new drug (IND) in neuropathic corneal pain and was granted Fast Track designation. OKYO expects to begin a 120-patient Phase 2b/3 multiple-dose clinical trial later in 2026.

What This FDA “Expanded Access” Authorization Really Means

When people hear “FDA authorization,” they often think it means a medicine is fully approved for sale. But expanded access (compassionate use) is different. It’s a pathway that can allow a patient with a serious or life-impacting condition to receive an investigational (not-yet-approved) treatment outside of a traditional clinical trial.

In this case, the FDA’s authorization is for a single patient. That’s important, because it signals the agency has reviewed key details—like the medical need, the patient’s lack of appropriate alternatives, and the product’s available safety information—to permit use under controlled conditions. It does not mean the therapy is proven for everyone, but it does mean the FDA considered the risk–benefit profile appropriate for this situation.

Expanded access programs typically include strict guardrails. Physicians must provide justification, comply with reporting requirements, and follow a protocol designed to protect the patient. From a patient perspective, this can feel like a lifeline—especially when standard options have been exhausted. From a biotech company’s perspective, it can be a meaningful validation that the unmet need is real and urgent.

Understanding Neuropathic Corneal Pain: A Condition That Can Be Debilitating

Neuropathic corneal pain is often described as chronic and severe eye pain that may not match what doctors can see on the surface of the eye. That mismatch can be incredibly frustrating for patients: the pain is very real, but standard exams may not always explain its intensity.

In simple terms, neuropathic corneal pain is thought to involve two major drivers:

• Immune-mediated inflammation that can irritate or sensitize ocular tissues
• Dysfunctional nerve signaling where corneal nerves misfire, amplify pain signals, or remain “stuck” in a hypersensitive state

This can lead to symptoms like burning, stinging, aching, light sensitivity, foreign-body sensation, and persistent discomfort that interferes with school, work, driving, reading, and sleep. The emotional impact can be huge, too—chronic pain can drain energy, attention, and motivation over time.

One of the most difficult realities is that there are no FDA-approved therapies specifically indicated for neuropathic corneal pain. Patients may be managed with a mix of approaches—such as lubricants, anti-inflammatory strategies, nerve pain approaches, or specialized contact lenses—depending on the case. But when severe pain persists, the need for targeted therapies becomes glaringly obvious.

Urcosimod: What OKYO Pharma Says Makes It Different

OKYO Pharma’s investigational therapy urcosimod is being studied as a potential treatment for neuropathic corneal pain with what the company describes as a dual mechanism of action. In plain language, “dual mechanism” means the therapy may work through two complementary pathways, rather than just one.

According to the company’s explanation, the dual approach may help address:

• Inflammatory pathways that can contribute to eye pain and ongoing irritation
• Nerve-related pathways involved in abnormal pain signaling

This matters because neuropathic corneal pain is not always a simple “dry eye” issue or a straightforward inflammation problem. For many patients, it can be a complex loop where inflammation and nerve dysfunction feed into each other. A therapy that aims to address both sides could be especially valuable—if clinical data continue to support that potential.

For this compassionate use authorization, the FDA has allowed urcosimod at a 0.05% dose for the single-patient expanded access setting. OKYO Pharma will provide the treatment under this FDA-authorized program.

Why This Compassionate Use Decision Is a Big Deal for Patients

Let’s not sugarcoat it: for someone with severe neuropathic corneal pain, daily life can become a grind. When you can’t rely on comfortable vision—when every blink hurts—ordinary routines become exhausting. In that context, compassionate use isn’t just paperwork; it can represent new hope when options are scarce.

This authorization matters for patients in at least five practical ways:

1) It Opens a Real Treatment Door—Now

A single patient with severe neuropathic corneal pain may now access a therapy that isn’t commercially available. That’s immediate, tangible impact.

2) It Highlights the Unmet Need

The FDA doesn’t authorize expanded access lightly. This decision helps underline what patients and specialists already know: there is a serious gap in approved treatments for neuropathic corneal pain.

3) It Encourages Attention to a “Hidden” Pain Condition

Because eye pain can be hard to measure on a standard exam, neuropathic corneal pain may be misunderstood. Coverage of expanded access can bring more awareness and validation.

4) It Reinforces the Importance of Specialized Care

Expanded access requests involve careful oversight and physician leadership. That can direct patients toward clinicians experienced in complex ocular pain.

5) It Provides a Pathway While Trials Progress

Clinical trials are essential, but they take time. Expanded access can sometimes bridge the gap for select cases, especially when no alternatives exist.

What It Means for OKYO Pharma’s Clinical Timeline

From a company strategy standpoint, this compassionate use authorization is aligned with OKYO Pharma’s broader plan to develop urcosimod as a therapy for neuropathic corneal pain through formal clinical trials.

Key program notes mentioned in the report include:

• FDA authorization for an IND in neuropathic corneal pain
• Fast Track designation, which is intended to support development of therapies for serious conditions with unmet medical needs
• A planned 120-patient Phase 2b/3 multiple-dose clinical trial expected to begin later in 2026

While compassionate use is not a substitute for large-scale trials, it can be an important signal—especially when it reflects strong medical rationale and the urgency of unmet need. For investors and observers, it can also suggest that the company is actively engaging with regulators and clinicians while pushing forward toward pivotal development steps.

Fast Track Designation: Why It Matters (Without the Hype)

Fast Track designation is often misunderstood as a “fast approval.” It’s not. But it can be meaningful because it’s designed to facilitate development and speed the review of drugs that treat serious conditions and fill an unmet medical need.

In practice, Fast Track can help with:

• More frequent communication with the FDA
• Potential eligibility for rolling review (submitting parts of an application as they’re ready)
• Better alignment on trial design and endpoints, when appropriate

For a condition like neuropathic corneal pain—where patients may have severe suffering and limited options—Fast Track aligns with the public health goal of supporting promising therapeutic development. Still, the ultimate decision rests on clinical evidence: the upcoming Phase 2b/3 program will be crucial.

How a 120-Patient Phase 2b/3 Trial Could Be Designed

OKYO Pharma has indicated it expects to begin a 120-patient Phase 2b/3 multiple-dose clinical trial later in 2026. While specific protocol details weren’t provided in the report, trials in ocular pain conditions commonly focus on a combination of:

• Patient-reported outcomes (because pain is experienced subjectively)
• Functional impact (light sensitivity, reading, screen tolerance, quality of life)
• Safety and tolerability (especially for chronic dosing)
• Biomarkers or clinical signs when relevant (inflammation measures, corneal nerve assessments in specialized settings)

A multiple-dose structure suggests the company is looking at sustained treatment rather than a one-time use, which fits the reality that neuropathic corneal pain is often chronic. A Phase 2b/3 approach can also indicate a program designed to both refine efficacy expectations and support later-stage decision-making—again, depending on the final statistical plan and endpoints.

Why Investors Are Watching Neuropathic Corneal Pain Programs Closely

Biotech investors often pay close attention to three ingredients: unmet need, clinical differentiation, and regulatory momentum. Neuropathic corneal pain sits at the intersection of all three.

Unmet Need Is Clear

The condition can be severe, chronic, and difficult to manage—and there are no FDA-approved therapies specifically indicated for it.

Differentiation Is the Big Question

Many eye therapies target inflammation, lubrication, or surface repair. A therapy that aims to address both inflammation and nerve dysfunction could stand out—if trial results confirm meaningful patient benefit.

Regulatory Momentum Can Reduce Uncertainty

Expanded access authorization and Fast Track designation can be viewed as indicators of active regulatory engagement and recognition of the medical need, even though they do not guarantee approval.

Safety and Patient Monitoring: What Expanded Access Typically Involves

Even in compassionate use, patient safety remains the core priority. Expanded access programs commonly involve:

• Eligibility confirmation (severity, lack of options, inability to join trials, etc.)
• Informed consent explaining risks and unknowns
• Physician oversight with scheduled follow-ups
• Adverse event reporting to track safety signals

Because urcosimod is investigational, broader safety and effectiveness must be established through controlled studies. Compassionate use is carefully bounded—one patient, one authorization, one structured approach.

What Comes Next: The Story to Watch Through 2026

This FDA authorization is a meaningful milestone, but it’s also part of a longer arc. Here’s what observers will likely watch next:

• Initiation of the Phase 2b/3 trial (timing, sites, enrollment pace)
• Trial design details (endpoints, dosing schedule, duration)
• Interim updates, if any are planned and appropriate
• Safety profile clarity as more patients receive treatment
• Regulatory interactions as the program advances

For patients, the most important question is simple: Will this therapy reduce severe eye pain safely and meaningfully? For clinicians, the question is how to position such a therapy within real-world care. For investors, the question is whether the data can support a differentiated product with a clear pathway to approval and adoption.

FAQs

1) What does “compassionate use” mean in this news?

Compassionate use is another term for expanded access. It can allow a patient with a serious condition to receive an investigational therapy outside a clinical trial when no comparable options exist.

2) Is urcosimod FDA-approved now?

No. The FDA authorization discussed here is for single-patient expanded access, not full approval. Urcosimod remains an investigational therapy.

3) What condition is urcosimod being used for in this authorization?

The authorization is for neuropathic corneal pain, a chronic condition involving severe eye pain linked to inflammation and dysfunctional nerve signaling.

4) What dose was authorized for this single-patient expanded access?

The FDA authorization allows urcosimod at a 0.05% dose for the compassionate use case described.

5) Why is this news important if it’s only for one patient?

Even a single-patient authorization can be meaningful because it highlights urgent unmet need, demonstrates regulatory engagement, and may provide a near-term option for someone with severe disease.

6) What is OKYO Pharma planning next for urcosimod?

OKYO expects to begin a 120-patient Phase 2b/3 multiple-dose clinical trial later in 2026. The company has also noted prior FDA IND authorization in this indication and Fast Track designation.

Conclusion

OKYO Pharma’s FDA authorization for single-patient compassionate use of urcosimod in neuropathic corneal pain is a notable development in a field where patients often struggle with severe symptoms and limited targeted options. It offers a carefully controlled pathway to treat a patient in need today, while also reinforcing the urgency behind the company’s broader clinical program. With a 120-patient Phase 2b/3 multiple-dose trial expected later in 2026, the next chapter will depend on what the data ultimately show: safety, real pain relief, and meaningful improvements in daily life.

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