Ilomastat

Summary

Ilomastat (GM6001) is a potent, broad-spectrum hydroxamic acid-based MMP inhibitor widely used as a research tool compound. It has been investigated for anti-tumor, anti-angiogenic, and anti-inflammatory applications. A topical ophthalmic formulation has been explored for corneal wound healing. It has not received regulatory approval for any systemic indication.

Mechanism of Action

Broad-spectrum inhibitor of matrix metalloproteinases (MMPs) including MMP-1, MMP-2, MMP-3, MMP-8, MMP-9, and MMP-14. Chelates the catalytic zinc ion in the MMP active site via its hydroxamic acid moiety, blocking collagen and extracellular matrix degradation. Also inhibits ADAM and TACE (ADAM-17) metalloprotease activity.

Routes of Administration

Goals & Uses

• Anti-angiogenesisOncology / VascularLow
• Corneal wound healing / persistent epithelial defectsOphthalmologyModerate
• Inflammatory disease suppressionInflammationLow
• Anti-tumor / cancer invasion suppressionOncologyLow
• Research tool: MMP pathway dissectionResearchHigh

Contraindications

• PregnancyPopulationHighPotential fetal risk or insufficient safety data
• Known hypersensitivity to hydroxamic acid derivativesAllergyHigh

Adverse Effects

• Musculoskeletal syndrome (MSS)MusculoskeletalCommon
• Nausea / GI disturbanceGastrointestinalUncommon
• Local ocular irritationOphthalmicUncommon

Drug Interactions

• Anticoagulants (e.g., warfarin)Low
• Other MMP inhibitors / metalloprotease inhibitorsModerate

Population Constraints

• Pediatric patientsAgeRelative
• Lactating womenReproductiveRelative
• Patients with hepatic or renal impairmentOrgan ImpairmentRelative

Regulatory Status

• European UnionInvestigationalNo EMA marketing authorization; used in research and limited investigational studies.
• United StatesInvestigationalStudied in Phase II clinical trials for corneal epithelial defects (topical formulation); not FDA-approved. Widely available as a research reagent.
• United KingdomInvestigationalNo MHRA approval; research use only.

Not approved by FDA, EMA, or MHRA for systemic clinical use. Has been evaluated in clinical trials for corneal epithelial defects (topical ophthalmic). Primarily used as a laboratory research reagent. Previous broader MMP inhibitor drug class faced setbacks in oncology trials due to musculoskeletal toxicity.

Evidence & Sources

No sources recorded yet.