LDE225 (Odomzo)
Other Medications · Approved since 2015
Description
LDE225 (sonidegib) is a small molecule hedgehog pathway inhibitor that blocks smoothened (SMO) protein signaling. In colorectal cancer, it targets tumors with specific mutations in hedgehog pathway components, particularly PTCH1 or SMO mutations. This targeted therapy is designed for patients whose tumors harbor these rare but actionable genetic alterations that drive hedgehog pathway activation.
Mechanism of Action
LDE225 binds to and inhibits the smoothened (SMO) protein, a key transmembrane component of the hedgehog signaling pathway. By blocking SMO, the drug prevents downstream activation of GLI transcription factors, ultimately suppressing hedgehog-mediated tumor growth and survival signals in cancer cells with pathway mutations.
Molecular Targets
Side Effects
Not all side effects are listed. Side effects vary by individual. Always consult your oncologist.
Clinical Trials
Quick Info
| Brand Name | Odomzo |
| Category | Other Medications |
 Status | FDA Approved |
| Administration | Oral |
| Year Approved | 2015 |
| Clinical Trials | 1 |
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Medical Disclaimer
This information is for educational purposes only and should not replace professional medical advice. Always consult your healthcare team before making treatment decisions.