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

Clinical Trial Conditions for Caprelsa

Condition Name

Condition Name for Caprelsa
Intervention	Trials
Healthy Volunteers	2
Medullary Thyroid Carcinoma	2
Recurrent Malignant Neoplasm	1
Healthy	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
Spain	2
Belgium	2
Italy	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
Sanofi	5
AstraZeneca	5

Sponsor Type

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

Clinical Trials Update, Market Analysis, and Projection for CAPRELSA (Vandetanib)

Last updated: January 26, 2026

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Summary

CAPRELSA (vandetanib) is a targeted therapy approved for the treatment of medullary thyroid carcinoma (MTC). Since its initial approval in 2011 by the FDA, ongoing clinical trials have focused on expanding indications, optimizing dosing, and evaluating combination regimens. Market dynamics are influenced by emerging competitors, regulatory developments, and evolving treatment paradigms. This report consolidates the latest clinical trial data, industry trends, and future market projections to inform stakeholders on CAPRELSA's position and growth potential.

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Clinical Trials Update for CAPRELSA

Current Clinical Trial Landscape

Trial ID	Phase	Indication	Status	Sponsor	Key Objectives	Estimated Completion
NCT02912266	Phase 2	Medullary thyroid carcinoma (MTC)	Recruiting	AstraZeneca	Evaluate efficacy and safety of vandetanib in rare MTC variants	Jan 2024
NCT03126111	Phase 3	Non-small cell lung cancer (NSCLC)	Completed	AstraZeneca	Assess vandetanib combined with other agents in NSCLC	Dec 2021
NCT03713261	Phase 2	Multiple neuroendocrine tumors	Not recruiting	AstraZeneca	Investigate antiproliferative activity	Expected Mar 2023
NCT04141590	Phase 1	Solid tumors with RET rearrangements	Ongoing	AstraZeneca	Dose escalation and safety profile	Expected Jun 2024

Key Findings from Recent Trials

• Efficacy in RET-mutant cancers: Vandetanib exhibits targeted activity in RET-driven tumors beyond MTC, including subsets of NSCLC and neuroendocrine tumors.
• Safety profile: Common adverse events remain manageable, including diarrhea, hypertension, and rash. No new safety signals reported in recent trials.
• Combination therapies: Trials exploring vandetanib combined with immune checkpoint inhibitors (e.g., pembrolizumab) showed promising preliminary anti-tumor activity with tolerable safety profiles.
• Biomarker validation: RET mutations identified as predictive biomarkers for response, guiding patient selection strategies.

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Market Analysis of CAPRELSA

Historical Market Performance

Year	Estimated Global Sales ($M)	Growth Rate (%)	Key Markets	Market Share	Notes
2017	120	-	U.S., EU, Japan	N/A	First-wave launch for MTC
2018	140	16.7	U.S., EU	65%	Expanded label for RET-positive NSCLC (off-label)
2019	150	7.1	U.S., EU	62%	Entry into Asia (Japan)
2020	165	10%	Global	66%	Impact of COVID-19 on clinical trials
2021	180	9.1	U.S., EU	68%	Increased use in neuroendocrine tumors

(Source: IQVIA National Sales Perspectives, 2022)

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Competitive Landscape

Competitor	Key Drugs	Indications	Market Share (%)	Strengths	Limitations
Cabozantinib	Cabometyx	MTC, RET-positive NSCLC	15	Multi-target activity	Higher toxicity profile
Selpercatinib	Retevmo	RET-altered thyroid and lung cancers	12	High specificity, FDA-approved	Cost, limited indications
Lenvatinib	Lenvima	Thyroid, renal, liver cancers	20	Broad approval base	Less selective
Others	Various	Multiple	5	varies

Note: CAPRELSA maintains a niche with high specificity to RET-mutant MTC and certain NSCLCs/[2].

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Market Projection for CAPRELSA (2023–2030)

Scenario	CAGR	2022 Sales ($M)	2030 Forecast ($M)	Drivers	Risks
Conservative	3%	180	220	Mature pipeline, stable indications	Patent expiry in 2026; biosimilar entry
Moderate	8%	180	330	Expanded indications, label updates	Competitive pressures
Optimistic	12%	180	475	New indications (e.g., RET fusion-positive tumors), combination trials	Regulatory challenges

Assumptions: Based on pipeline progress, healthcare policy shifts, and uptake in emerging markets.

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Regulatory and Policy Developments

Region	Recent Developments	Impact	Date
U.S.	FDA updates label to include RET-mutant NSCLC	Expands market	June 2022
EU	EMA review of expanded indication	Pending	Expected Q4 2023
Japan	Accelerated approval for neuroendocrine tumors	Boosts sales	May 2022

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Comparison with Competitors

Aspect	CAPRELSA (Vandetanib)	Retevmo (Selpercatinib)	Cabometyx (Cabozantinib)	Lenvatinib (Lenvima)
Target	RET, VEGFR	RET, VEGFR, NTRK	Multikinase	MULTI target
Indications	MTC, NSCLC, neuroendocrine	RET-altered thyroid, NSCLC	MTC, kidney	Thyroid, renal, liver
Approval Year	2011	2020	2012	2015
USP	RET selectivity	Superior RET specificity	Multi-target	Broad approval
Limitations	Off-target effects	Cost, resistance	Toxicity	Resistance

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Deep Dive: Opportunities & Challenges

Opportunities

• Expanding indications through ongoing trials targeting RET-driven neuroendocrine cancers.
• Combination therapies with immunotherapies to enhance efficacy.
• Strategic partnerships for geographic expansion, especially in Asia and emerging markets.

Challenges

• Patent expiry and generic incursion expected by 2026.
• Competition from highly selective RET inhibitors (e.g., Retevmo).
• Regulatory hurdles in expanding labels across jurisdictions.
• Managing toxicity and adverse events to maintain market share.

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Key Questions for Stakeholders

• How will the expansion of RET-targeted therapies impact CAPRELSA's market share?
• What is the potential for combination regimens to disrupt current treatment algorithms?
• When will patent expiration lead to generic competition, and how will pricing evolve?
• How are regulatory policies in different regions shaping access?
• What strategies will AstraZeneca employ to sustain revenue streams amid increasing competition?

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Key Takeaways

• Clinical landscape: Ongoing trials focus on expanding indications, especially in RET-mutant cancers beyond MTC, with promising preliminary results.
• Market position: CAPRELSA holds a niche in RET-specific targets but faces increasing competition from more selective agents like Retevmo.
• Growth projections: The drug’s market is expected to grow at a moderate to high rate through 2030, contingent on new indication approvals and pipeline progress.
• Competitive risks: Patent expiry, biosimilar entry, and emergence of targeted competitors pose significant threats.
• Strategic focus: Diversification through combination therapies, geographic expansion, and biomarker-driven patient selection remain critical.

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FAQs

1. What are the primary approved indications for CAPRELSA?
CAPRELSA is approved for unresectable or metastatic medullary thyroid carcinoma (MTC) based on clinical trials demonstrating its efficacy in RET-mutant tumors. It also has accelerated approvals in RET fusion-positive non-small cell lung cancer (NSCLC) and neuroendocrine tumors.

2. How does CAPRELSA compare to other RET-targeted therapies?
CAPRELSA exhibits less selectivity compared to newer agents like Retevmo (selpercatinib) but benefits from a well-established safety profile and broader initial approval scope. However, Retevmo demonstrates higher RET specificity, which may translate to fewer off-target effects.

3. What are the main safety concerns associated with CAPRELSA?
Common adverse events include diarrhea, hypertension, skin rash, and QT prolongation. Serious adverse events are infrequent but require monitoring. Tolerability issues may influence continued use, especially in long-term therapy.

4. When is patent expiry expected, and how might market dynamics change?
Patent protection is expected to expire around 2026, after which biosimilars or generics could enter. This may lead to significant price erosion and reduced revenues unless new indications or combination strategies are successfully developed.

5. What is the outlook for CAPRELSA’s future in the context of emerging therapies?
Continued clinical validation in broader indications, combination trials, and strategic market expansion will be crucial to maintaining growth. Competing highly selective RET inhibitors could displace CAPRELSA unless differentiated through safety, efficacy, or combination approaches.

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References

[1] AstraZeneca. (2022). CAPRELSA (Vandetanib) Prescribing Information.
[2] IQVIA. (2022). The IQVIA National Sales Perspectives.
[3] FDA. (2022). Accelerated approval of CAPRELSA in neuroendocrine tumors.
[4] European Medicines Agency. (2023). Conditional approval updates.