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Odomzo (sonidegib)

Odomzo® (sonidegib) is used to treat certain cases of locally advanced basal cell carcinoma (BCC). Locally advanced BCC is cancer that still confined to the skin tumor, but it is large, recurrent, or aggressive.1

Odomzo is used to treat locally advanced BCC that:2

  • Has recurred (come back) after surgery or radiation therapy
  • Cannot be treated with surgery or radiation therapy

Odomzo does not come in generic form. Odomzo is similar to Erivedge® (vismodegib), another drug that is approved to treat certain kinds of advanced basal cell carcinoma.

How does Odomzo work?

Odomzo blocks a cell signaling pathway called Hedgehog.2 This pathway has a role in the development of hair follicles and glands. It also helps to control skin growth.

Key players in the Hedgehog pathway are two receptors called PTCH1 (“Patched”) and SMO (“Smoothened”).3 In normal cells, PTCH1 acts like an off-switch for SMO. It prevents SMO from sending signals for cell division and survival.

In about 40% of BCC tumors, PTCH1 is mutated.4 Mutated PTCH1 does not turn SMO off, so SMO starts sending out signals. This leads to uncontrolled cell growth and survival.

Odomzo works by blocking the SMO receptor.2 It is called a targeted therapy, because it targets one feature that makes cancer cells different from normal cells.

How effective is Odomzo?

Odomzo was studied in 194 people with locally advanced BCC. Of those people, 66 were given the 200 mg once daily dose that has been approved for use.

The researchers used a measure called “objective response rate” to determine if the medication worked.2 Objective response rate has to do with how the tumor changes during treatment. It includes several factors, including tumor size and ulceration. (Ulceration is when the top layer of cells breaks down. This causes a hole to form in the skin, and the tissue below shows through.5) Response to treatment can be partial or complete. Complete response means no BCC was found on a follow-up biopsy. Partial response is when the tumor shrinks or the ulceration heals. The objective response rate does not measure whether you—as a whole patient—have improved.

After treatment with 200 mg of Odomzo:2

  • 5% of patients with locally advanced BCC had a complete response.
  • 53% of patients with locally advanced BCC had a partial response.

When Odomzo works, it seems to work for quite a while in most people. In the latest published report from July 2016, very few responders had progressed or died.6 This good news means the researchers have not been able to determine the median duration of response.

How do I take Odomzo?

Before taking Odomzo, read the Medication Guide that comes in the package. Follow your doctor’s instructions for dosing and duration.

Odomzo comes as a pink capsule that is taken by mouth.2 Take Odomzo once a day on an empty stomach. Take it at least 1 hour before or 2 hours after a meal.

You will continue taking Odomzo until the cancer progresses or the side effects are unacceptable.2

What are the side effects of Odomzo?

Muscle spasms and muscle pain are common side effects of Odomzo.2 In rare cases, taking Odomzo increases your risk of a muscle injury called rhabdomyolysis. Rhabdomyolysis can lead to kidney damage. Symptoms of muscle problems and kidney damage include:

  • New or worsening muscle spasms
  • Pain or tenderness
  • Dark urine
  • Low urine output (production)

Nearly all patients experience at least one side effect.7 Most are mild or moderate. Serious adverse reactions occurred in 18% of study participants.2 The most common side effects of Odomzo are:2

  • Muscle spasms and muscle or joint pain
  • Hair loss
  • Taste changes, loss of appetite, weight loss
  • Fatigue
  • Gastrointestinal problems (nausea, vomiting, diarrhea or constipation)

Women may stop having a menstrual period while taking Odomzo. It is not known whether this change is permanent.

Who should not take Odomzo?

Odomzo can cause still birth or severe birth defects.2 Pregnant women should not take Odomzo. Women who could become pregnant should use birth control during treatment and for at least 20 months after the last dose. Do not breastfeed while taking Odomzo or for 20 months after the last dose.

It is not known whether Odomzo is present in semen.2 Men should always use a condom during sex with a partner who is or could become pregnant. Use a condom during treatment and for at least 8 months after the last dose of Odomzo. Do not donate semen during this time.

Tell your doctor about any medical conditions you have. In particular, tell your doctor if you have a history of a muscle disorder called rhabdomyolysis or myopathy. Blood tests can be done to check for muscle problems and kidney function. Your doctor will do this test before and during treatment with Odomzo.

What precautions are needed when taking Odomzo?

During treatment with Odomzo and for 20 months after the last dose:2

  • Do not donate blood or blood products.
  • Women should use effective birth control.
  • Women should not breastfeed.

During treatment with Odomzo and for at least 8 months after the last dose, men whose sexual partners are pregnant or could become pregnant should use a condom.

Written by: Sarah O'Brien | Last reviewed: October 2018.
  1. Migden MR, Guminski A, Gutzmer R, et al. Treatment with two different doses of sonidegib in patients with locally advanced or metastatic basal cell carcinoma (BOLT): a multicentre, randomised, double-blind phase 2 trial. Lancet Oncol. 2015;16:716-728.
  2. Odomzo® [prescribing information]. East Hanover, NJ: Novartis Pharmaceuticals Coporation; July 2015.
  3. My Cancer Genome. Hedgehog signaling. Accessed January 18, 2016 at:
  4. Rudin C. Molecular profiling of basal cell carcinoma (Updated July 15, 2015). My Cancer Genome. Accessed January 6, 2017 at:
  5. National Cancer Institute. Staging. Accessed February 7, 2017 at:
  6. Dummer R, Guminski A, Gutzmer R, et al. The 12-month analysis from Basal Cell Carcinoma Outcomes with LDE225 Treatment (BOLT): A phase II, randomized, double-blind study of sonidegib in patients with advanced basal cell carcinoma. J Am Acad Dermatol. 2016;75:113-125.
  7. NCCN Clinical Practice Guidelines in Oncology. Basal cell skin cancer. Version 1.2017. Published October 3, 2016.