OKYO Pharma Limited announced the FDA has granted Fast Track designation to its lead asset, a neuropathic corneal pain (NCP) therapeutic—just shy of 2 months since filing a request.
First, a Fast Track designation refresher.
What it is: As part of the FDA program that expedites the development and review process of investigational drug candidates intended to treat, modify, reverse, or cure a serious or life-threatening condition—and fulfill unmet medical needs.
Note: This program also includes Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough designations (BTD)
- However: A key distinction of FTD is that (unlike BTD), this can be requested with non-clinical data and/or preliminary clinical evidence.
Now tell me about this NCP candidate.
Formerly referred to as OK-101, urcosimod is a lipid-conjugated chemerin peptide antagonist of the ChemR23 G-protein-coupled receptor (GPCR).
- For a reminder of why the ChemR23 G-protein is critical, click here (hint: it plays a key role in managing ocular inflammation and immune responses).
Its current investigational indications:
- Allergic conjunctivitis
- Dry eye disease (DED)
- NCP
- Uveitis
So why target NCP?
To start: There are currently no FDA-approved and commercially available treatments for the severe (and often misdiagnosed) chronic disease.
Even further: Standard therapies used to manage NCP—including steroids, opioids (for extreme cases), and nonsteroidal anti-inflammatory drugs (NSAIDs)—are largely only effective for the short term, and with variable success.
Got it. And how does urcosimod treat this disease?
The candidate utilizes OKYO’s proprietary membrane-anchorded-peptide (MAP) platform, allowing it to develop a novel long-acting drug candidate that’s administered via topical application.
Specifically: This asset features a built-in lipid “anchor” formulated to prevent washout and increase the amount of time it (the drug) can reside within its ocular environment (i.e., the eye).
Interesting … now, didn’t we just see new data on it?
Indeed we did—just last month, in fact.
The gist of that: OKYO announced positive findings supporting urcosimod’s long-term stability for NCP treatment.
More specifically: The company reported that daily administration of urcosiomd (via single-use ampoules) was shown to be stable for +2.5 years as well as minimize contamination risks.
The numbers: Based on two varying doses of the solution, the following high percentages of stability were demonstrated:
- 0.05% urcosimod (94.8%)
- 0.1% urcosimod (97.1%)
Remind me why long-term stability is crucial.
It’s a key component urcosimod needs to demonstrate and include in a potential new drug application (NDA) submission package to the FDA for marketing approval.
The drug’s successful demonstration of this was noted by OKYO CEO Gary S. Jacob, PhD, as “a critical positive step” in meeting the federal agency’s chemistry, manufacturing, and controls (CMC) guidance for NDA submissions.
And before this? What did clinical data find?
Data collection for this NCP indication is still underway via a 12-week phase 2 trial (topline data is expected in Q3 2025).
But for its DED indication: A first-in-human phase 2 study found urcosimod to:
- Demonstrate favorable safety and tolerability
- Lead to statistically significant improvements in dry eye symptoms
Nice! So with this new Fast Track designation, what’s next?
With this designation comes several advantages for urcosimod, including more frequent meetings and communications with the FDA to go over urcosimod’s:
- Development plans (including proposed clinical trial designs and biomarker use)
- Potential eligibility for accelerated approval and priority review (if criteria are met)
- Rolling review of its NDA
- This enables OKYO to submit complete sections of its NDA in stages rather than waiting until all sections are completed (as is standard, non-expedited practice)
Keep in mind: The FDA already approved urcosimod’s investigational new drug application (IND) for NCP—and DED.