Toceranib

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Toceranib

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Medically reviewed

Laura Arias

Last updated on 22/12/2025

This page provides general, reference-level information compiled from official medical sources. It is not a substitute for professional medical advice, diagnosis, or treatment. For decisions about your health, please consult a qualified healthcare professional.

Overview of Toceranib

Property Description
Active Ingredient Toceranib phosphate (SU11654)
Form Oral tablets (film-coated)
Pharmacological Class Multi-targeted Tyrosine Kinase Inhibitor (TKI), Antineoplastic Agent
General Purpose Managing uncontrolled cellular proliferation
Origin Synthetic small molecule (Indolinone derivative)

What Type of Drug is Toceranib?

Toceranib is a synthetic small molecule drug primarily classified as a Multi-targeted Tyrosine Kinase Inhibitor (TKI) and an Antineoplastic Agent. The active pharmaceutical ingredient is Toceranib phosphate (SU11654), a derivative built upon the Indolinone chemical structure.

This medication functions as a targeted therapy, which differentiates it from non-specific chemotherapeutics by inhibiting the activity of specific Receptor Tyrosine Kinase (RTK) enzymes. Its development is clinically recognized for providing a valuable, non-traditional medical approach to managing certain progressive conditions, and it was the first TKI approved specifically for veterinary use.

What is the Composition and Form of Toceranib?

Toceranib is a single-ingredient product, most commonly supplied as a film-coated oral tablet for administration by mouth. The composition consists solely of the active ingredient, Toceranib phosphate, combined with the necessary pharmaceutical excipients used to form the solid dosage.

Its formulation includes an integral tablet coating, a key feature that requires the tablet to be administered whole, without being split or crushed, to maintain its integrity. This controlled composition allows for flexibility in use, as the medicine is also available as compounded capsules or an oral liquid form, depending on specific patient needs.

What is the General Purpose of Toceranib?

The general purpose of Toceranib is to manage conditions characterized by uncontrolled cellular proliferation by interfering with the cell's key growth signaling pathways. As a TKI, its action involves simultaneously disrupting the function of several crucial RTKs, including VEGFR2, PDGFR, and Kit/c-kit.

By acting against these molecular targets, the drug provides both a direct anti-proliferative effect on the abnormal cells and antiangiogenic activity—which hinders the development of the new blood vessels needed to sustain the affected cell population. This targeted molecular intervention serves to stabilize the progression of the condition by limiting the growth commands and restricting the nutrient supply to the proliferating cells.

Regulatory References

  1. FDA Approval History

What side effects are possible with Toceranib?

Possible Side Effects and Safety Information

Official regulatory documents classify the possible adverse reactions of toceranib based on body systems and frequency, with the majority of common effects classified as Very Common, meaning they may affect more than 1 in 10 individuals.

Very Common Adverse Reactions

Reactions most frequently documented in regulatory labeling often involve the gastrointestinal system and blood cell counts. Gastrointestinal disturbances include diarrhea, vomiting, anorexia, blood in faeces, and hemorrhagic diarrhea. Hematological effects are defined by decreases in specific blood cell types, such as neutropenia (low white blood cells) and thrombocytopenia (low platelets). General systemic effects like lethargy, weight loss, pruritus (itching), and lameness are also commonly listed. Laboratory changes may include elevated alanine aminotransferase (ALT) levels, indicating potential effects on the liver.

Serious Adverse Reactions and Safety Restrictions

Official prescribing information highlights the potential for severe adverse events, including the risk of gastrointestinal perforation, a serious and sometimes fatal complication. Thromboembolism (blood clot formation) is also documented as a serious safety concern. The regulatory label includes specific safety constraints, such as an increased risk of gastrointestinal ulceration when toceranib is used concurrently with Non-Steroidal Anti-Inflammatory Drugs (NSAIDs). Use must be discontinued prior to surgery due to the associated risk of vascular dysfunction. Furthermore, toceranib is contraindicated and should not be used in breeding, pregnant, or lactating individuals.

Overdose and Emergency Response

Toceranib Overdose and when to seek help — official regulatory information

Toceranib overdose can be fatal and is officially considered an emergency, as the drug has a narrow margin of safety, meaning even a small amount over the prescribed dose may result in toxic effects.

Documented Overdose Presentations

Official documents describe overdose manifestations involving the gastrointestinal, hematopoietic, and systemic systems. Signs of an overdose may include:

  • Gastrointestinal: Vomiting, diarrhea, loss of appetite, or signs of bleeding (such as unexplained bruising, bright red blood in vomit or stools, or black tarry stools).
  • Systemic/Musculoskeletal: Low energy (lethargy), limping, or muscle cramps.

Required Emergency Action

In the event of a suspected or witnessed overdose, immediate medical attention is required. Regulatory labeling emphasizes the need to immediately contact a medical professional or a poison control center for advice.

Accidental Human Ingestion

Accidental ingestion by a person may cause gastrointestinal discomfort, specifically vomiting or diarrhea. If this occurs, the person must seek medical advice immediately and show the drug's package insert or label to the physician. Pregnant or nursing individuals are advised to be particularly cautious, as accidental ingestion may have adverse effects on pregnancy.

This information is based on governmental regulatory documents that define the overdose profile and emergency-seeking conditions, without providing further clinical advice or interpretation.

Therapeutic Uses of Toceranib

Quick Facts: Toceranib Uses

  • Approved for use in: Dogs.
  • Primary Application: Management of specific cutaneous mast cell tumors.
  • Goal: To help manage tumor growth and recurrence.

Toceranib is an established, prescription-only veterinary medication indicated for the management of certain cancers in dogs. Its primary approved use is for the treatment of Patnaik grade II or III, recurrent, cutaneous mast cell tumors (MCTs) that occur on the skin.

This treatment is used in dogs whether or not the regional lymph nodes are affected by the tumor. The medication is an option for veterinarians seeking to address the recurrence of these specific cutaneous tumors. Toceranib may contribute to the overall therapeutic strategy in dogs diagnosed with this condition.

As with all prescription treatments, the use of toceranib should be discussed and determined by a qualified veterinarian, considering the specific health needs of the animal and standard veterinary oncology practices.

Eligibility and Restrictions for Use

Eligibility Map: Who Can and Cannot Use Toceranib

This section summarizes the official population eligibility and non-eligibility for Toceranib phosphate, based strictly on government regulatory documents.

Eligibility Status Applicable Population/Condition
Allowed Use Dogs with Patnaik grade II or III, recurrent, cutaneous mast cell tumors.
Absolute Contraindication Dogs used for breeding, or pregnant or lactating bitches.
Absolute Contraindication Dogs with known hypersensitivity to the active substance or evidence of gastrointestinal bleeding.
Unestablished Safety Dogs less than 24 months of age or those weighing less than 5 kg (11 lbs).
Conditional Use Dogs suffering from hepatic disease (must be used with caution).
Time Restriction Use must be discontinued for at least 3 days prior to surgery.

Official Eligibility Statements:

  • Toceranib is formally contraindicated in patients with a reproductive status of breeding, pregnancy, or lactation.
  • Regulatory documents state that use is not evaluated (safety is not established) in patients under 24 months of age or below 5 kg of weight.
  • The use is restricted to cautionary status in patients with pre-existing hepatic disease.

The overall eligibility profile is defined by contraindications that prohibit use in specific reproductive and acute clinical states, alongside limitations for juvenile patients where safety data is officially unestablished.

What should I know about interactions with other medicines?

Interactions with other medicines and products

The official regulatory profile for toceranib phosphate outlines specific pharmacokinetic and pharmacodynamic interactions that require attention. No medicinal product combination is formally classified as absolutely contraindicated in the product labeling.

Pharmacokinetic (Exposure-Altering) Interactions

Toceranib is extensively metabolized in the liver. Co-administration with certain medicinal products that are strong inhibitors of the CYP3A4 enzyme family may increase the circulating plasma concentration of toceranib. This change in drug exposure is officially noted as a potential consequence of impaired hepatic clearance. The label also advises that the effect of drugs capable of inducing liver enzymes should be considered, as this may alter toceranib's elimination.

Pharmacodynamic (Additive Toxicity) Interactions

A documented interaction exists with Non-steroidal anti-inflammatory drugs (NSAIDs). Concurrent use is associated with an increased risk of gastrointestinal ulceration or perforation due to an additive toxicity effect.

Administration and Contextual Restrictions

  • Procedural Restriction: Toceranib must be discontinued for at least 3 days before performing surgery. This mandatory timing requirement is specified to ensure proper vascular homeostasis.
  • Food: The medicine may be administered with or without food, as regulatory data indicates no clinically significant difference in exposure based on the fed or fasted state.
  • Hepatic Status: Caution is required when used in patients with hepatic disease, as the drug’s metabolism and subsequent clearance are highly dependent on liver function.

Mechanism of Action

Toceranib phosphate is a multi-targeted tyrosine kinase inhibitor that modulates dysregulated cellular proliferation signals through a combined two-pronged mechanism of action.

Targeted Blockade of Cell Growth Signals

Toceranib directly interferes with the biological processes that drive cell survival by acting as a competitive inhibitor of the KIT (c-Kit) receptor tyrosine kinase. By binding to the receptor's internal ATP site, the drug prevents the essential process of autophosphorylation, thereby switching off the internal signaling cascades (like the MAPK pathway) that drive cell proliferation and division. This action leads to the cell cycle arrest and apoptosis (programmed death) of the targeted cells.

Disruption of Vascular Supply and Maturation

The drug also modulates the systemic environment by inhibiting the Vascular Endothelial Growth Factor Receptor 2 (VEGFR2) and Platelet-Derived Growth Factor Receptor beta (PDGFRbeta). This dual inhibition restricts the signaling required for endothelial cells to form new blood vessels (angiogenesis) and for the vessels to achieve stability. The resulting physiological consequence is an anti-angiogenic state that restricts the signaling required for nutrient supply pathways to the abnormal cell mass.

Dosage and Administration Information

How Toceranib is Used: Official Administration Guidelines

Toceranib phosphate is administered strictly via the oral route as a film-coated tablet. The medication is formulated to be swallowed whole and the tablets must not be broken, split, or crushed to ensure the integrity of the controlled dosing.


Dosing and Frequency

The initial recommended dosage is standardized at 3.25 mg/kg of body weight, with the medicine administered on an every-other-day schedule. Administration is not conditional on feeding, as the dose may be given with or without food. Should a scheduled dose be missed, a double dose must not be given.


Procedural Monitoring and Adjustments

Standard use involves a protocol of mandatory veterinary re-assessment to determine the appropriate dose and duration of treatment. This professional review is conducted approximately weekly for the first six weeks of use, and then approximately every six weeks thereafter. Adjustments, if necessary, utilize a standardized decrement of 0.5 mg/kg, allowing for dosage reduction down to a minimum of 2.2 mg/kg. Furthermore, the medicine has not been evaluated for safety in subjects under 2 years of age or those weighing less than 5 kg.

Recent Clinical Evidence

Research Evidence / Overview of Studies for Toceranib

Evidence for Use in Recurrent Cutaneous Mast Cell Tumors

Research examining Toceranib for recurrent cutaneous mast cell tumors (MCTs) in dogs includes placebo-controlled, double-blind, randomized clinical trials (RCTs). This type of research, a randomized trial with a control group, was applied in the evaluation of the medicine. These studies were applied in client-owned dogs with a specific diagnosis of Grade II or III MCTs. The trials' primary endpoints were to monitor Objective Response Rate (ORR) (tumor regression and disappearance) and measure the Time to Tumor Progression (TTP).

Studies monitored Objective Response Rate measurements reported in the drug-treated group and compared them with measurements from the placebo group. Research monitored TTP over defined time intervals to explore patterns of tumor stabilization. Studies examined whether dogs with tumors possessing c-kit gene mutations were associated with different reported Objective Response Rate measurements than dogs with wild-type c-kit.

Research into Other Canine Solid Tumors

Research has explored the use of Toceranib for a heterogeneous range of other solid tumors in dogs. Studies in this area mainly consisted of Phase 1 dose-escalation studies and descriptive retrospective analyses of clinical records, which are considered lower-level evidence than RCTs. Studies examined parameters such as the Clinical Benefit Rate (CBR), which monitored tumor stabilization or response (partial/complete), as well as survival time endpoints like Progression-Free Survival (PFS).

Studies reported measurements of Clinical Benefit Rate and Progression-Free Survival across these varied tumor types, including apocrine gland anal sac adenocarcinomas and thyroid carcinomas. Findings describe patterns observed in the studies, but these generally lack the confirmation of a large-scale, randomized, controlled trial.

What Remains Unclear or Under Investigation

Evidence highlights what is known—and what is still uncertain—about this medicine. A primary limitation is the lack of randomized, controlled evidence for tumor types other than the specific cutaneous mast cell tumors. Long-term effects are not fully established to characterize the durability of objective response beyond intermediate follow-up periods. Research is ongoing to fully understand the mechanisms of eventual non-responsiveness to the treatment that was observed in some study populations.

Frequently Asked Questions (FAQ)

Common questions about Toceranib (FAQ)


Q: Is hair loss a typical side effect associated with Toceranib?

A: The primary official documents list itching (pruritus) as a 'Very Common' adverse reaction, but hair loss (alopecia) is not included in the list of most frequently documented effects.


Q: Is it true that Toceranib can affect blood pressure?

A: High blood pressure (systemic hypertension) is noted in official reports as a possible adverse effect associated with Toceranib. Clinical studies have indicated that treatment can be associated with increases in systolic blood pressure.


Q: Do side effects usually start right away, or do they appear after taking it for a while?

A: Due to the potential for side effects to appear early in treatment, the official monitoring protocol requires mandatory re-assessment by a professional on a frequent basis for the initial weeks. This schedule is in place to quickly detect and manage potential adverse reactions, particularly changes in blood counts and liver enzymes.


Q: If Toceranib is working, how will I generally know or feel a change?

A: Official information indicates that effectiveness is assessed through scheduled professional re-assessments. Assessment involves monitoring for signs of response, such as changes in the abnormal cell masses or stabilization, which are clinical endpoints used in studies.


Q: How does Toceranib interact with grapefruit juice, if at all?

A: The drug is metabolized in the liver by the CYP3A4 enzyme family. The official regulatory documents do not provide specific instructions or warnings regarding the concurrent use of grapefruit juice.


Q: What should a patient do if they experience skin rash while on Toceranib?

A: Official information lists pruritus (itching) as a very common adverse reaction. Any new or worsening skin reaction observed, such as a rash, should be discussed with the prescribing professional for evaluation.


Q: What are the risks if Toceranib is taken at the same time as blood thinners?

A: The official label documents an increased risk of thromboembolism (blood clot formation) as a serious adverse reaction of the drug. Concurrent use with blood thinners or other medicines affecting clotting requires consideration by the prescribing professional.


Q: Can I travel while taking Toceranib and are there any precautions?

A: Official instructions specify that the drug must be stored at Controlled Room Temperature in its original, tightly closed, and locked container. Special precautions are required for the disposal of waste (feces, urine, vomit) generated during treatment due to the cytotoxic nature of the drug. These requirements apply regardless of location.


Q: If I have a history of heart problems, does that prevent me from using Toceranib?

A: The official product information lists thromboembolism (blood clot formation) as a serious safety concern. Official information indicates that a history of heart or vascular issues is a factor that must be considered by the prescribing professional.


Q: What kind of specialist usually prescribes Toceranib?

A: Toceranib phosphate is a prescription drug restricted by law to use by or on the order of a licensed veterinarian. Official information states that a licensed veterinary professional is required to prescribe and manage this treatment.


Q: How is Toceranib different from other common treatments for the same condition?

A: Toceranib is classified as a Multi-targeted Tyrosine Kinase Inhibitor (TKI), which is a type of targeted therapy. This mechanism is described as differentiating it from traditional non-specific chemotherapeutics by specifically interfering with the growth signals and blood supply pathways of abnormal cells.


Q: Are there any common over-the-counter medicines or supplements that can interfere with Toceranib?

A: Official warnings exist regarding concurrent use with Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) due to the increased risk of gastrointestinal bleeding. Additionally, drugs that strongly inhibit or induce the liver's CYP3A4 enzymes can potentially alter the concentration of Toceranib in the body.


Q: Can certain foods or drinks change how Toceranib works?

A: The medicine can be given with or without food, as regulatory data indicates no clinically significant difference in overall exposure based on the fed or fasted state. However, the drug's metabolism via the CYP3A4 liver enzyme means that foods or drinks that strongly affect this enzyme could potentially alter its effect.


Q: Is Toceranib meant to cure a condition, or is it meant to manage symptoms?

A: The general purpose of the drug is to manage conditions characterized by uncontrolled cellular proliferation by interfering with growth signals and restricting blood supply. Its action is focused on stabilizing the progression of the condition.


Q: Are there currently ongoing clinical trials for new uses of Toceranib?

A: While the drug has an approved use, research has been conducted and continues to explore its potential application for a variety of other solid tumor types. Findings from these exploratory studies are described in the research evidence section.


Q: Why is it necessary for doctors to check bloodwork while a person is taking Toceranib?

A: Bloodwork monitoring is necessary because the official safety information lists changes in blood cell counts and liver function as very common side effects. Specifically, decreases in white blood cells (neutropenia), decreases in platelets (thrombocytopenia), and elevated liver enzymes (ALT) are commonly monitored.


Q: What is the meaning of the specific safety class Toceranib belongs to?

A: Toceranib is classified as an Antineoplastic Agent, and its disposal requires special precautions for cytotoxic medicinal waste. This classification indicates that the drug is designed to interfere with actively dividing cells, necessitating careful handling and disposal.


Q: How is Toceranib processed by the body and eventually eliminated?

A: Toceranib is extensively metabolized in the liver. The process of its elimination, or clearance, is highly dependent on healthy liver function. Official warnings note that the effect of drugs that alter liver enzyme activity should be considered.


Q: Is the mechanism of action of Toceranib fully understood?

A: While the main mechanism of action (inhibiting key receptors like KIT and VEGFR2) is well-defined, research is ongoing. Evidence highlights that the mechanisms of eventual non-responsiveness to the treatment observed in some studies are still under investigation.

How should Toceranib be stored and disposed of?

Official Storage and Disposal Requirements

Tocerabib phosphate tablets must be stored at Controlled Room Temperature, specifically between 20 C and 25 C (68 F and 77 F), with permitted excursions up to 30 C. The medication must be kept in its original, tightly closed container and should be stored locked up to comply with safety regulations.

Storage Classification Requirement
Temperature Controlled Room Temperature (20 C to 25 C)
Packaging Keep in original container, tightly closed
Child Safety Keep out of the sight and reach of children and pets

Disposal procedures are mandatory due to the cytotoxic nature of the drug. Protective gloves must be worn when handling the tablets, or any contaminated materials such as the feces, urine, or vomit of treated animals. All contaminated waste must be placed in a sealed plastic bag before general disposal. Unused product requires disposal according to special precautions for cytotoxic medicinal waste, avoiding discharge into water courses or drains.

Attention! Always consult to a doctor or pharmacist before using pills or medicines.

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