Repifermin
Also known as: FGF-10 truncated, KGF-2, recombinant human keratinocyte growth factor, rHuKGF, rHuKGF-2, SY-002
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Summary
Repifermin is a recombinant, truncated form of human keratinocyte growth factor‑2 (FGF‑10), classified as a growth‑factor biologic. It is being investigated for conditions involving epithelial injury, such as chemotherapy‑induced oral mucositis and chronic skin ulcers. The compound is administered intravenously, subcutaneously, or topically, but it has not received regulatory approval for any indication.
Mechanism of Action
Repifermin binds the FGFR2b (keratinocyte growth factor receptor) on epithelial cells, activating downstream MAPK and PI3K‑Akt pathways that promote cell proliferation, migration, and differentiation. This epithelial‑specific signaling leads to thickening of mucosal linings and accelerates re‑epithelialisation of damaged surfaces while sparing non‑epithelial tissues.
What the Research Shows
Early clinical work shows repifermin reduces the incidence of moderate‑to‑severe oral mucositis in patients undergoing autologous stem‑cell transplantation, with 50–64% of treated patients experiencing Grade 2‑4 mucositis versus 100% on placebo. A randomized, double‑blind trial in venous leg ulcers demonstrated faster wound closure, with a higher proportion of patients achieving ≥75% reduction in ulcer area compared with placebo, especially in smaller, newer ulcers. In contrast, a dose‑escalation study in active ulcerative colitis found no statistically significant improvement in remission rates. Pharmacokinetic studies in monkeys and healthy volunteers reveal dose‑proportional exposure, high tissue binding, reversible mucosal changes, and low immunogenicity. A recent network meta‑analysis of wound‑healing agents listed repifermin (60–120 µg/cm²) among many comparators, noting modest cure rates and a low infection signal, but emphasized the need for larger trials.
Reported Benefits
Evidence from controlled trials indicates that repifermin can lessen the severity of chemotherapy‑related oral mucositis and accelerate closure of chronic venous ulcers, likely through enhanced epithelial proliferation. The drug has been consistently reported as well tolerated, with adverse‑event rates comparable to placebo in the studied populations.
Limitations of the Evidence
The mucositis and ulcer studies involved relatively small sample sizes and short follow‑up, limiting confidence in long‑term efficacy. No benefit was observed in ulcerative colitis at doses up to 50 µg/kg, and the systematic review placed repifermin low among wound‑healing agents, highlighting the scarcity of large, definitive trials. Consequently, regulatory approval has not been achieved, and optimal dosing regimens remain undefined.
Safety Considerations
Across Phase I/II and ulcer trials, repifermin was generally well tolerated; the frequency of common and severe adverse events did not differ from placebo. In monkeys, reversible mucosal thickening occurred without gastric involvement, suggesting epithelial specificity. Human pharmacokinetic data showed no drug accumulation with daily dosing and minimal immunogenicity. No serious safety signals have emerged, but long‑term safety remains uncharacterized.
How It Is Administered
Repifermin has been delivered intravenously (single or daily infusions), subcutaneously (daily injections), and topically (gel or cream formulations). Clinical studies used IV doses ranging from 1 to 50 µg/kg and topical concentrations of 60–120 µg per cm² applied over several weeks. Formulations are investigational and not commercially available.
Routes of Administration
Goals & Uses
- Prevention of oral mucositisOncology Supportive CareModerate
- Epithelial repairRegenerative MedicineLow
- Wound healing (venous leg ulcers)Tissue RepairModerate
- Gastrointestinal mucosal injury repairGastroenterologyLow
- Accelerated wound healingDermatologyLow
- Oral mucositis preventionOncology Supportive CareLow
- Inflammatory bowel diseaseGastroenterologyLow
Contraindications
- Known hypersensitivity to the drugAllergyHigh
- Active malignancy at treatment siteOncologyHigh
- Known hypersensitivity to KGF-2 or FGF analogsAllergyHigh
- Active malignancy with high FGFR2b expressionOncologyModerate
Adverse Effects
- EdemaFluid BalanceUncommonSwelling from fluid retention
- RashDermatologicUncommonSkin eruption or discoloration
- Local Injection Site ReactionsDermatologicalCommon
- Taste alteration (dysgeusia)SensoryCommon
- HypotensionCardiovascularRareLow blood pressure
- Erythema / skin rashDermatologicCommon
- FeverSystemicUncommonElevated body temperature
Drug Interactions
- Other growth factorsLow
- Other growth factor therapiesLow
- Concurrent chemotherapyModerate
Population Constraints
- Pediatric patientsAgeRelative
- Lactating mothersReproductiveRelative
- Pregnant womenReproductiveRelative
- Patients with epithelial malignanciesOncologyRelative
Regulatory Status
- European UnionInvestigationalNo EMA marketing authorization
- United StatesInvestigationalNot FDA‑approved; clinical trials ongoing
- United KingdomInvestigationalNot authorized for clinical use
Never received FDA or EMA approval. Development was halted after Phase II/III trials failed to demonstrate sufficient efficacy for the primary indications pursued.
Evidence & Sources
- Journal ArticleModerateFreytes CO, et al.2004-01-01T00:00:00.000000Z
- Journal ArticleHighWu Y, et al.2024-01-01T00:00:00.000000Z
- Journal ArticleModerateRobson MC, et al.2001-01-01T00:00:00.000000Z
- Journal ArticleModerateSandborn WJ, Targan SR2002-01-01T00:00:00.000000Z
- Journal ArticleModerateSung C, et al.2002-01-01T00:00:00.000000Z
- Journal ArticleModerateSandborn WJ, et al.2003-01-01T00:00:00.000000Z
Frequently Asked Questions
What types of conditions is repifermin being studied for?
Repifermin is under investigation for diseases where epithelial damage is central, such as chemotherapy‑induced oral mucositis, chronic venous leg ulcers, and, experimentally, inflammatory bowel disease. Early trials have shown benefit in mucositis and skin ulcer healing, but not in ulcerative colitis.
How does repifermin differ from other growth‑factor therapies?
It is a truncated, recombinant version of human keratinocyte growth factor‑2 that selectively activates FGFR2b on epithelial cells, leading to targeted mucosal thickening without affecting non‑epithelial tissues. This receptor specificity distinguishes it from broader growth‑factor preparations.
Is repifermin approved for clinical use?
No. Repifermin remains an investigational biologic. It has not received FDA or EMA approval for any therapeutic indication, and its use is confined to controlled clinical research settings.
What are the main safety concerns with repifermin?
Clinical trials have reported a safety profile similar to placebo, with no increase in severe adverse events. Animal studies noted reversible mucosal thickening, and human data show low immunogenicity. Long‑term safety and effects of higher doses have not been fully evaluated.
How is repifermin administered in studies?
Researchers have used intravenous infusions (1–50 µg/kg), daily subcutaneous injections (up to 750 µg/kg in monkeys), and topical applications (60–120 µg per cm²) applied over weeks. The exact formulation and schedule depend on the specific trial protocol.
What is Repifermin?
Repifermin is a recombinant, truncated form of human keratinocyte growth factor‑2 (FGF‑10), classified as a growth‑factor biologic. It is being investigated for conditions involving epithelial injury, such as chemotherapy‑induced oral mucositis and chronic skin ulcers. The compound is administered intravenously, subcutaneously, or topically, but it has not received regulatory approval for any indication.
What is Repifermin used for?
Repifermin is educationally associated with: Prevention of oral mucositis, Epithelial repair, Wound healing (venous leg ulcers), Gastrointestinal mucosal injury repair, Accelerated wound healing, Oral mucositis prevention, Inflammatory bowel disease. Educational only — not medical advice.
How is Repifermin administered?
Recorded routes of administration: Intravenous, Subcutaneous, Topical.
What are the potential side effects of Repifermin?
Reported adverse effects include: Edema, Rash, Local Injection Site Reactions, Taste alteration (dysgeusia), Hypotension, Erythema / skin rash, Fever. This list is not exhaustive — consult a qualified clinician.
Who should avoid Repifermin?
Recorded contraindications: Known hypersensitivity to the drug, Active malignancy at treatment site, Known hypersensitivity to KGF-2 or FGF analogs, Active malignancy with high FGFR2b expression. Consult a qualified clinician before use.