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These drugs target parts of skin cancer cells that make them different from normal skin cells. Targeted drugs work differently from standard chemotherapy drugs. They may work sometimes when chemotherapy drugs don’t. They may also have less severe side effects. Doctors are still learning the best way to use these drugs to treat skin cancers.

Hedgehog pathway inhibitors

Examples of targeted drugs include vismodegib (Erivedge) and sonidegib (Odomzo), which can be used to treat some advanced or recurrent basal cell skin cancers.

It’s very rare for basal cell cancers to reach an advanced stage, but if they do, these cancers can be hard to treat. Most basal cell cancers have mutations (changes) in genes that are part of a cell signaling pathway called hedgehog. The hedgehog pathway is crucial for the development of the embryo and fetus and is important in some adult cells, but it can be overactive in basal cell cancer cells. These drugs target a protein in this pathway.

These drugs are capsules taken once a day. For basal cell cancers that have spread or come back after surgery or other local treatments, these drugs have been shown to help shrink tumors in some people, although it’s not yet clear if they help people live longer.

Side effects can include muscle spasms, joint pain, hair loss, fatigue, problems with taste, poor appetite and weight loss, nausea and vomiting, itchy skin, diarrhea, and constipation. These drugs can also cause women to stop having their periods.

Because the hedgehog pathway affects fetal development, these drugs should not be taken by women who are pregnant or could become pregnant. It is not known if they could harm the fetus if taken by a male partner. Anyone taking these drugs should use reliable birth control during and for some time after treatment.


Last Medical Review: 04/01/2016
Last Revised: 05/10/2016