CLINICAL TRIALS PROFILE FOR CAPRELSA

All Clinical Trials for Caprelsa

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00459121 ↗	Vandetanib, Carboplatin, and Paclitaxel in Treating Patients With Stage I, Stage II, or Stage III Non-Small Cell Lung Cancer That Can Be Removed by Surgery	Terminated	National Cancer Institute (NCI)	Phase 2	2007-07-01	RATIONALE: Vandetanib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor. Drugs used in chemotherapy, such as carboplatin and paclitaxel, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving vandetanib together with chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. PURPOSE: This phase II trial is studying how well giving vandetanib together with carboplatin and paclitaxel works in treating patients with stage I, stage II, or stage III non-small cell lung cancer that can be removed by surgery.
NCT00459121 ↗	Vandetanib, Carboplatin, and Paclitaxel in Treating Patients With Stage I, Stage II, or Stage III Non-Small Cell Lung Cancer That Can Be Removed by Surgery	Terminated	Barbara Ann Karmanos Cancer Institute	Phase 2	2007-07-01	RATIONALE: Vandetanib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor. Drugs used in chemotherapy, such as carboplatin and paclitaxel, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving vandetanib together with chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. PURPOSE: This phase II trial is studying how well giving vandetanib together with carboplatin and paclitaxel works in treating patients with stage I, stage II, or stage III non-small cell lung cancer that can be removed by surgery.
NCT00514046 ↗	Vandetanib to Treat Children and Adolescents With Medullary Thyroid Cancer	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2007-07-20	Background: - Medullary thyroid carcinoma (MTC) is common in people with a genetic disorder called multiple endocrine neoplasia (MEN). - Vandetanib is an experimental drug that blocks a defective protein receptor (rearranged during transfection (RET) receptor) found on the surface of cancer cells in people with MEN. It is thought that this protein is a primary cause of MTC in people with MEN. Objectives: - To study the activity of Vandetanib in children and adolescents with MEN-related MTC by measuring the change in tumor size, in blood levels of proteins produced the tumor (calcitonin and carcinoembryonic antigen (CEA) and in tumor-related diarrhea. - To determine the safety and tolerability of Vandetanib in children and adolescents. - To study how the body handles Vandetanib in children and adolescents. - To determine the effect of Vandetanib on the survival of children and adolescents with MTC. Eligibility: -Children and adolescents 5 to 18 years of age with MTC whose tumor cannot be surgically removed or has grown back after treatment or has metastasized (spread beyond the thyroid gland). Design: - Patients take Vandetanib once a day in 28-day cycles. The first patients enrolled in the study are started on a low dose of Vandetanib to determine tolerability. - Patients have periodic blood tests, electrocardiograms, and blood pressure measurements to look for side effects of Vandetanib. - Blood tests and imaging scans (magnetic resonance imaging (MRI), computed tomography (CT), bone and octreoscan) are done every 8 weeks for the first 32 weeks of treatment and then every 16 weeks for the duration of the treatment period. - Patients who have tumor-related diarrhea keep a daily record of the number and consistency of bowel movements.
NCT00923247 ↗	A Targeted Phase I/II Trial of ZD6474 (Vandetanib; ZACTIMA) Plus the Proteasome Inhibitor, Bortezomib (Velcade ), in Adults With Solid Tumors With a Focus on Hereditary or Sporadic, Locally Advanced or Metastatic Medullary Thyroid Cancer (MTC)	Terminated	National Cancer Institute (NCI)	Phase 1/Phase 2	2009-02-19	Background: - The combination of anti-cancer drugs vandetanib (given orally) and bortezomib (given intravenously) has not been used in humans. However, both drugs have been studied separately. Bortezomib has been approved by the U.S. Food and Drug Administration (FDA) for treating multiple myeloma and mantle cell lymphoma, while vandetanib is still under investigation pending FDA approval. - Both bortezomib and vandetanib are under investigation for use in treating certain kinds of cancer. Researchers hope that the combination of these two drugs will be more effective than either of them alone. Objectives: - To determine if the combination of vandetanib and bortezomib will decrease the amount of the cancer and, if it does, to determine how long the response will last. - To determine any side effects that may occur with this combination of treatments. - To determine what doses of each drug are well tolerated and safe when given together. - To study genetic mutations in tumors to better understand how tumors grow and how these drugs interact with the tumor. Eligibility: - Patients 18 years of age and older with solid tumors that cannot be surgically removed and have either recurred or shown further growth. The tumor(s) must be able to be evaluated by X-ray, MRI (magnetic resonance imaging), and CT (computerized tomography) scanning. - Patients who have been diagnosed with medullary thyroid cancer will participate in Phase II of the study. Design: - Tumor samples may be taken at the start of the study for research purposes. - Phase I: Patient groups will be treated on an outpatient basis with vandetanib and bortezomib, given at increasing doses over four different levels to determine the maximum tolerated dose calculated by height and weight: - Doses will be given on Days 1, 4, 8, and 11 for each 28-day cycle. - Two additional levels (Level 1A and Level 1B) may be included in the study, depending on side effects at various levels. - Phase II: Patients with medullary thyroid cancer will be divided into two groups, with two patients in Group A for every one patient in Group B. No placebo will be involved in this study. - Group A: Patients will be treated with vandetanib and bortezomib at the maximally tolerated dose of the Phase I study. - Group B: Patients will be treated with bortezomib alone. - A second tumor sample may be taken. In patients with thyroid cancer, the second biopsy will be done at the 6-week evaluation (approximately 42 days after beginning). In patients with cancer other than thyroid cancer, the second biopsy will be obtained on Day 4 of either the first or second cycle, after the bortezomib infusion. - The effects of the drugs will be studied through blood samples and CT scans taken during and after various drug cycles.
NCT01539655 ↗	Study in Healthy Volunteers to Assess Effect of Omeprazole and Ranitidine on the Pharmacokinetics of Vandetanib	Completed	Sanofi	Phase 1	2012-02-01	Study in healthy volunteers to assess effect of omeprazole and ranitidine on the pharmacokinetics of vandetanib
NCT01544140 ↗	Study in Healthy Volunteers to Assess the Pharmacokinetics of Midazolam Administered Alone and in Combination With Vandetanib	Completed	Sanofi	Phase 1	2012-04-01	The purpose of this study in healthy volunteers is to assess the Pharmacokinetics (PK) of Midazolam administered alone and in combination with Vandetanib.

Clinical Trial Conditions for Caprelsa

Condition Name

Condition Name for Caprelsa
Intervention	Trials
Healthy Volunteers	2
Medullary Thyroid Carcinoma	2
Metastatic Breast Cancer	1
Plasma [AUC(0-672)]	1

Condition MeSH

Condition MeSH for Caprelsa
Intervention	Trials
Thyroid Neoplasms	6
Carcinoma, Neuroendocrine	4
Thyroid Diseases	4
Lung Neoplasms	3

Clinical Trial Locations for Caprelsa

Trials by Country

Trials by Country for Caprelsa
Location	Trials
United States	20
France	4
Italy	2
United Kingdom	2
Spain	2

Trials by US State

Trials by US State for Caprelsa
Location	Trials
Kansas	4
Maryland	4
Michigan	2
Pennsylvania	1
Oregon	1

Clinical Trial Progress for Caprelsa

Clinical Trial Phase

Clinical Trial Phase for Caprelsa
Clinical Trial Phase	Trials
Phase 3	1
Phase 2	5
Phase 1/Phase 2	3

Clinical Trial Status

Clinical Trial Status for Caprelsa
Clinical Trial Phase	Trials
Completed	9
Terminated	3
Active, not recruiting	3

Clinical Trial Sponsors for Caprelsa

Sponsor Name

Sponsor Name for Caprelsa
Sponsor	Trials
National Cancer Institute (NCI)	6
AstraZeneca	5
Sanofi	5

Sponsor Type

Sponsor Type for Caprelsa
Sponsor	Trials
Industry	13
Other	12
NIH	6

Caprelsa (vandetanib): Clinical Trials Update, Market Analysis, and Projection

Last updated: April 28, 2026

Caprelsa (vandetanib) is a small-molecule VEGFR/EGFR inhibitor for advanced metastatic or unresectable medullary thyroid cancer (MTC) and for locally advanced or metastatic NSCLC with activating or sensitizing EGFR mutations (per label language in major markets). The product’s commercial trajectory is dominated by (i) the duration of prior-line therapies in MTC, (ii) MTC competitive dynamics driven by selpercatinib and pralsetinib, and (iii) NSCLC competition across EGFR TKI and immuno-oncology sequencing. Current activity in the landmark development program is largely in legacy datasets, with ongoing efforts focused on combination strategies, resistance biology, and label expansion refinements rather than a new late-stage pivotal registration path.

What do the latest Caprelsa clinical trials show?

MTC program: treatment durability and combination exploration

The pivotal basis for Caprelsa in MTC is established in earlier randomized and supportive trials (notably in metastatic/unresectable MTC). Current trial activity in the public domain is largely characterized by:

• Phase 1 to 2 studies assessing combinations (targeted plus targeted, or targeted plus cytotoxic agents), and
• Biomarker-linked follow-on studies focusing on resistance pathways (VEGFR/EGFR axis signaling, downstream MAPK/PI3K activity) and disease heterogeneity.

Commercial implication: For MTC, where there is no single curative option, Caprelsa’s value proposition depends on position in treatment sequencing and on tolerability relative to newer targeted agents that are biomarker-driven.

NSCLC program: legacy EGFR-targeted positioning

Caprelsa has an established label footprint in NSCLC for EGFR-mutant disease in some jurisdictions based on prior clinical evidence. Public-facing clinical work has since shifted toward:

• alternative EGFR strategies (third-generation TKIs and combination regimens),
• resistance-informed combinations, and
• toxicity-management studies in broader EGFR-mutant populations.

Commercial implication: NSCLC growth for Caprelsa is constrained by modern standard-of-care use patterns in EGFR-mutant disease, where newer TKIs and sequencing strategies generally reduce addressable share.

Observed status of late-stage registration

No new Caprelsa late-stage pivotal registration dataset is the controlling factor in current market modeling; the product is primarily supported by existing evidence and real-world use rather than by a new Phase 3 readout driving label expansion in the near term.

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How big is Caprelsa’s market today?

Market segmentation and revenue drivers

Caprelsa’s revenue pools align to two labeled indications:

Indication	Patient setting	Primary revenue drivers	Main headwinds
MTC	metastatic or unresectable; later-line durable treatment	ability to maintain disease control; dosing continuity	targeted competitors in RET-driven pathways; earlier adoption of newer agents in some geographies
NSCLC (EGFR-mutant)	locally advanced/metastatic; EGFR-driven disease	fit within certain sequencing frameworks	competitive displacement by newer EGFR TKIs and IO-first/combination standards

Competitive landscape

• MTC: Competitive displacement risk is elevated because MTC biology overlaps with actionable pathways in tumors with RET alterations; newer RET-selective agents have captured attention and share where appropriate testing workflows exist.
• NSCLC: Caprelsa’s EGFR-directed role faces structural erosion as newer EGFR TKIs and evidence-based sequencing evolve.

Net effect for projections: Caprelsa is modeled as a mature, label-dependent product with revenue constrained by competitive entry and life-cycle dynamics rather than sustained growth from new trials.

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What is the commercial projection for Caprelsa?

Projection framework

This projection is built from product life-cycle assumptions that typically govern mature oncology brands:

• Indication-specific net demand tied to incidence and treatment penetration,
• Share erosion from entrants and guideline changes,
• Price and access dynamics across major markets,
• Formulary and reimbursement stability dependent on local guideline positions.

Base case: revenue trend

• Short term (0 to 2 years): revenue remains relatively stable to mildly down as competitive displacement continues.
• Medium term (2 to 5 years): gradual decline as RET-driven alternatives expand and NSCLC standard-of-care continues to shift.
• Long term (5+ years): steeper erosion risk if multiple geographies further align MTC/NSCLC sequencing away from vandetanib.

Scenario table (directional, for planning)

Scenario	MTC trend	NSCLC trend	Net revenue outcome
Base case	slow decline	moderate decline	steady-to-declining revenue
Conservative	sharper decline from competitive uptake	steeper displacement	faster revenue erosion
Upside	better than expected persistence in MTC lines	slower NSCLC share loss	slower decline

Actionable conclusion: Caprelsa is best treated as a declining cash-flow asset with pockets of durability in MTC where prior-line usage persists and where treatment sequencing and tolerability decisions favor continuation.

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What should business decision-makers watch next?

Clinical and regulatory watch items

1. Ongoing combination studies that can extend real-world use through better tolerability or efficacy signals.
2. Biomarker and resistance research that may influence patient selection logic in practice.
3. Label and safety communications that can alter adherence and persistence (dose modifications, AE management).

Commercial watch items

1. RET testing penetration in MTC, which shifts patient assignment toward RET-selective regimens where actionable alterations exist.
2. Guideline sequencing in EGFR-mutant NSCLC, which determines the proportion of patients offered earlier-line TKIs vs subsequent therapy positioning.
3. Tender and reimbursement cycles that affect net price in major markets.

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Key Takeaways

• Caprelsa is a mature oncology brand whose commercial performance is driven by label-linked use in MTC and EGFR-mutant NSCLC rather than by a new late-stage pivotal clinical program.
• MTC is the longer durability pool, but share erosion risk rises with the spread of RET-selective targeted therapies and testing workflows.
• NSCLC demand is structurally pressured by evolving EGFR standard-of-care and sequencing shifts, limiting upside.
• Near-term revenue is modeled as stable-to-declining, with medium and long-term trends trending down under continued competitive displacement.

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FAQs

1) What are Caprelsa’s primary labeled indications?

Caprelsa is approved for advanced metastatic or unresectable medullary thyroid cancer (MTC) and for locally advanced or metastatic NSCLC with activating or sensitizing EGFR mutations in the relevant jurisdictions per label language.

2) Why has competitive pressure increased for Caprelsa in MTC?

Because MTC treatment pathways increasingly incorporate molecularly targeted options and broader biomarker testing, which shifts eligible patients away from older multi-kinase approaches where newer selective agents fit treatment algorithms.

3) Is Caprelsa’s clinical development currently centered on late-stage readouts?

Public clinical activity is largely consistent with ongoing exploratory and combination work rather than a new late-stage pivotal registration driver dominating near-term market modeling.

4) How does NSCLC sequencing affect Caprelsa demand?

EGFR-mutant NSCLC management increasingly follows newer EGFR-directed standards and combination/IO strategies that reduce the proportion of patients treated with older agents, especially outside specific sequencing niches.

5) What is the most important variable for revenue persistence?

Treatment persistence and patient selection in MTC versus continued displacement by newer targeted regimens, plus NSCLC share loss driven by guideline sequencing.

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References

[1] European Medicines Agency. Caprelsa (vandetanib) product information. EMA.
[2] U.S. Food and Drug Administration. Caprelsa (vandetanib) prescribing information. FDA.
[3] PubMed. Vandetanib clinical trials in medullary thyroid cancer and EGFR-mutant NSCLC (indexed records). National Library of Medicine.
[4] National Comprehensive Cancer Network. Guidelines for thyroid cancer and NSCLC (vandetanib-related historical positioning; updates across versions). NCCN.