Sonidegib

Sonidegib is an inhibitor of the Hh pathway. Sonidegib binds to and inhibits Smoothened, a transmembrane protein involved in Hh signal transduction.

• ODOMZO (sonidegib) is indicated for the treatment of adult patients with locally advanced basal cell carcinoma (BCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy. ODOMZO is a hedgehog pathway inhibitor indicated for the treatment of adult patients with locally advanced basal cell carcinoma (BCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy.

• None. None.contraindicated

Recommended dosage: 200 mg orally once daily taken on an empty stomach, at least 1 hour before or 2 hours after a meal. ( 2.2 ) 2.1 Important Safety Information Verify the pregnancy status of females of reproductive potential prior to initiating ODOMZO [see Use in Specific Populations (8.1 , 8.3 )] . 2.2 Recommended Dosage The recommended dosage of ODOMZO is 200 mg taken orally once daily on an empty stomach, at least 1 hour before or 2 hours after a meal, administered until disease progression or unacceptable toxicity [see Clinical Pharmacology (12.3) ]. Obtain serum creatine kinase (CK) levels and renal function tests prior to initiating ODOMZO in all patients [see Dosage and Administration (2.2) and Warnings and Precautions (5.2) ]. If a dose of ODOMZO is missed, resume dosing with the next scheduled dose. 2.3 Dosage Modifications for Adverse Reactions Interrupt ODOMZO for Severe or intolerable musculoskeletal adverse reactions. First occurrence of serum CK elevation between 2.5 and 10 times upper limit of normal (ULN). Recurrent serum CK elevation between 2.5 and 5 times ULN. Resume ODOMZO at 200 mg daily upon resolution of clinical signs and symptoms. Permanently discontinue ODOMZO for Serum CK elevation greater than 2.5 times ULN with worsening renal function. Serum CK elevation greater than 10 times ULN. Recurrent serum CK elevation greater than 5 times ULN. Recurrent severe or intolerable musculoskeletal adverse reactions.

Embryo-Fetal Toxicity: Advise patients not to donate blood or blood products during treatment with ODOMZO and for at least 20 months after the last dose. ( 5.1 ) Musculoskeletal Adverse Reactions: Obtain serum creatine kinase (CK) and creatinine levels prior to initiating therapy, periodically during treatment, and as clinically indicated. Temporary dose interruption or discontinuation of ODOMZO may be required based on the severity of musculoskeletal adverse reactions. ( 2.2 , 5.2 ) Premature fusion of the epiphyses ( 5.3 , 8.4 ) 5.1 Embryo-Fetal Toxicity ODOMZO can cause embryo-fetal death or severe birth defects when administered to a pregnant woman. In animal reproduction studies, sonidegib was embryotoxic, fetotoxic, and teratogenic at maternal exposures below the recommended human dose of 200 mg [see Use in Specific Populations (8.1) ] . Females of Reproductive Potential Verify pregnancy status of females of reproductive potential prior to initiating ODOMZO treatment. Advise pregnant women of the potential risk to a fetus. Advise females to use effective contraception during treatment with ODOMZO and for at least 20 months after the last dose [see Use in Specific Populations (8.3) ] . Males Advise male patients with female partners to use condoms, even after a vasectomy, during treatment with ODOMZO and for at least 8 months after the last dose to avoid potential drug exposure in pregnant females or females of reproductive potential [see Use in Specific Populations (8.3) ] .

The following clinically significant adverse reactions are described elsewhere in the labeling: Musculoskeletal Adverse Reactions [see Warnings and Precautions (5.2) ] . The most common adverse reactions occurring in ≥10% of patients are muscle spasms, alopecia, dysgeusia, fatigue, nausea, musculoskeletal pain, diarrhea, decreased weight, decreased appetite, myalgia, abdominal pain, headache, pain, vomiting, and pruritus. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sun Pharmaceutical Industries, Inc. at 1-800-406-7984 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice. The safety of ODOMZO was evaluated in BOLT, a randomized, double-blind, multiple cohort trial in which 229 patients received ODOMZO at either 200 mg (n=79) or 800 mg (n=150) daily. The frequency of common adverse reactions including muscle spasms, alopecia, dysgeusia, fatigue, nausea, decreased weight, decreased appetite, myalgia, pain, and vomiting was greater in patients treated with ODOMZO 800 mg as compared to 200 mg. The data described below reflect exposure to ODOMZO 200 mg daily in 79 patients with locally advanced BCC (laBCC; n=66) or metastatic BCC (mBCC; n=13) enrolled in BOLT. Patients were followed for at least 18 months unless discontinued earlier.

Lactation: Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on its mechanism of action and data from animal reproduction studies, ODOMZO can cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology (12.1) ]. There are no available data on the use of ODOMZO in pregnant women. In animal reproduction studies, oral administration of sonidegib during organogenesis at doses below the recommended human dose of 200 mg resulted in embryotoxicity, fetotoxicity, and teratogenicity in rabbits (see Data). Teratogenic effects observed included severe midline defects, missing digits, and other irreversible malformations. Advise pregnant women of the potential risk to a fetus. Report pregnancies to Sun Pharmaceutical Industries, Inc. at 1-800-406-7984. In the U.S. general population, the estimated background risk of major birth defects is 2-4% and of miscarriage in clinically recognized pregnancies is 15-20%. Data Animal Data Daily oral administration of sonidegib to pregnant rabbits resulted in abortion, complete resorption of fetuses, or severe malformations at ≥ 5 mg/kg/day (approximately 0.05 times the recommended human dose based on AUC). Teratogenic effects included vertebral, distal limb and digit malformations, severe craniofacial malformations, and other severe midline defects.

Metabolism

Sonidegib exhibited dose-proportional increases in the area under the curve (AUC) and the maximal concentration (C max ) over the dose range of 100 mg to 400 mg, but less than dose-proportional increases at doses greater than 400 mg. Steady-state was reached approximately 4 months after starting ODOMZO and the estimated accumulation at steady-state was 19-fold.

• Jul 24, 2015NDANDA205266Sun Pharm

1. 1Muscle Spasms19513%
2. 2Alopecia1177.9%
3. 3Death1057.1%
4. 4Off Label Use1047.0%
5. 5Fatigue986.6%
6. 6Product Use Issue724.9%
7. 7Nausea714.8%
8. 8Product Dose Omission Issue694.7%
9. 9Therapy Cessation684.6%
10. 10Ageusia644.3%
11. 11Blood Creatine Phosphokinase Increased644.3%
12. 12Asthenia624.2%
13. 13Weight Decreased624.2%
14. 14Decreased Appetite614.1%
15. 15Drug Ineffective583.9%

The 10 most recently updated of 51 ClinicalTrials.gov registrations naming Sonidegib as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• AD HOC Trial: Artificial Intelligence-Based Drug Dosing In Hepatocellular Carcinoma
  Recruiting · Phase 2 · Interventional · 12 enrolled · University of Florida
  NCT05669339updated 2026-06-04
• Tailored Sonidegib Schedule After Complete Response in BCC
  Active not recruiting · Phase 2 · Interventional · 21 enrolled · Gruppo Oncologico del Nord-Ovest
  NCT04806646updated 2026-06-01
• Sonidegib and Pembrolizumab in Treating Patients With Advanced Solid Tumors
  Active not recruiting · Phase 1 · Interventional · 36 enrolled · Mayo Clinic
  NCT04007744updated 2026-03-18
• Post-authorization Safety Study on the Long Term Safety of Sonidegib in Patients With Locally Advanced Cell Carcinoma
  Completed · Observational · 323 enrolled · Sun Pharmaceutical Industries Limited
  NCT04066504updated 2026-03-18
• Phase IB Trial of LDE225 and Paclitaxel in Recurrent Ovarian Cancer
  Completed · Phase 1 · Interventional · 15 enrolled · University of Alabama at Birmingham
  NCT02195973updated 2026-02-09
• Blue-Light Photodynamic Therapy and Sonidegib for Multiple Basal Cell Carcinomas
  Recruiting · Phase 1 · Interventional · 20 enrolled · Nathalie Zeitouni
  NCT06623201updated 2026-01-29
• A Study to Evaluate Neoadjuvant Sonidegib Followed by Surgery or Imiquimod in the Management of Basal Cell Carcinoma
  Completed · Phase 2 · Interventional · 16 enrolled · Melanoma Institute Australia
  NCT03534947updated 2025-12-16
• Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma
  Terminated · Phase 2 · Interventional · 20 enrolled · European Organisation for Research and Treatment of Cancer - EORTC
  NCT04402073updated 2025-10-23
• Radiotherapy by Sonic Hedgehog Pathway Inhibitors in Basal Cell Carcinoma
  Recruiting · Phase 2 · Interventional · 82 enrolled · University Hospital, Lille
  NCT05561634updated 2025-09-30
• Sonidegib in Adult Patients With Locally Advanced Basal Cell Carcinoma (laBCC) in China
  Completed · Phase 4 · Interventional · 160 enrolled · Jemincare
  NCT06880848updated 2025-09-24

Frequently asked questions

How does Sonidegib work?

Sonidegib is an inhibitor of the Hh pathway. Sonidegib binds to and inhibits Smoothened, a transmembrane protein involved in Hh signal transduction.

What is Sonidegib used for?

According to FDA labeling, Sonidegib carries indications including: ODOMZO (sonidegib) is indicated for the treatment of adult patients with locally advanced basal cell carcinoma (BCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy. ODOMZO is a hedgehog pathway inhibitor indicated for the treatment of adult patients with locally advanced basal cell carcinoma (BCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Sonidegib?

Sonidegib is classified as Hedgehog pathway inhibitors, Hedgehog Pathway Inhibitor, Smoothened Receptor Antagonists.

What are the brand names for Sonidegib?

Sonidegib is marketed under brand names including Odomzo.

What are the contraindications for Sonidegib?

Sonidegib labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.