Vismodegib
Hedgehog pathway inhibitors
Hedgehog pathway inhibitorsHigh alert
An error with this medicine can cause serious harm: double checks and protocols are used.Pregnancy: contraindicated
Contraindicated in pregnancy (EU label). Vismodegib can cause embryo-fetal death or severe birth defects: a pregnancy test is done first, women use contraception during and for…
The first oral Hedgehog pathway inhibitor: blocks Smoothened in advanced basal cell carcinoma. Causes severe birth defects.
Quick facts
- Group
- Hedgehog pathway inhibitors
- Target
- Smoothened (SMO) receptor
- Used for
- Metastatic basal cell carcinoma
- Key effects
- Muscle spasms · Hair loss · Loss or change of taste
- Monitor
- Pregnancy test before starting · Weight and nutrition
- ATC
- L01XJ01
No doses: the clinician sets them by the current guideline.
Mechanism of action
The structure shows vismodegib in the transmembrane pocket of human Smoothened.
In basal cell carcinoma the Hedgehog pathway is stuck on, usually because the brake protein PTCH1 is lost. Smoothened then signals freely and GLI transcription factors drive growth. Vismodegib binds Smoothened and shuts the signal down, so GLI target genes are switched off and the tumour shrinks.
- Vismodegib binds Smoothened (SMO)
- Hedgehog signal stops and GLI factors switch off
- Basal cell carcinoma growth stops
Indications
- Metastatic basal cell carcinoma, or locally advanced disease that recurred after surgery or cannot be treated with surgery or radiation. Basal cell carcinoma →
Adverse effects
Common
- Muscle spasms
- Hair loss
- Loss or change of taste
- Weight loss and decreased appetite
- Fatigue, nausea, diarrhoea, missed periods
Contraindications
- Hypersensitivity to vismodegib
- Pregnancy (EU label)
Key interactions
- Prescription and over-the-counter drugs, vitamins and herbal products
- Reviewed before starting.
What is monitored
- Pregnancy test before starting in women who could conceive
- Weight and nutrition (taste loss)
- Muscle symptoms
Administration and safety
- Oral capsules swallowed whole, not split, chewed or crushed.
- No blood donation during treatment and for months after; men do not donate semen.
- Not shared with anyone.
Pregnancy and breastfeeding
Contraindicated in pregnancy (EU label). Vismodegib can cause embryo-fetal death or severe birth defects: a pregnancy test is done first, women use contraception during and for months after treatment, men use condoms, and blood donation is avoided.
Nursing care
- Confirm a negative pregnancy test and reliable contraception; men use condoms even after vasectomy.
- Teach that taste loss and hair loss are common and support nutrition.
- Teach to report a missed period or unprotected sex at once.
- No breastfeeding during and for months after treatment.
Pharmacokinetics
- Absorption
- Oral capsules once a day, with or without food.
Test yourself
Think of the answer, then tap the question to see it.
Which protein does vismodegib block?
Smoothened (SMO), in the Hedgehog pathway.
Which genetic change usually drives basal cell carcinoma?
Loss of the PTCH1 brake, which leaves Smoothened active.
Which two adverse effects of vismodegib are very common?
Muscle spasms and loss of taste (also hair loss and weight loss).
Connections
← Back to the class: Hedgehog pathway inhibitors
Educational content on how drugs work; not a prescribing guide and no substitute for a health professional's advice. No doses are given.
Sources: Vismodegib: MedlinePlus Drug Information (MedlinePlus (U.S. National Library of Medicine), 2016); EMA: product information for vismodegib (European Medicines Agency, 2025); RCSB PDB - 5L7I: Structure of human Smoothened in complex with Vismodegib (RCSB Protein Data Bank, 2026); ATCDDD - ATC/DDD Index (WHO Collaborating Centre for Drug Statistics Methodology, 2026).
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Representative model, for educational purposes only.