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
Cmax	1
Metastatic Malignant Neoplasm	1

Condition MeSH

Condition MeSH for CAPRELSA
Intervention	Trials
Thyroid Neoplasms	6
Carcinoma, Neuroendocrine	4
Thyroid Diseases	4
Carcinoma, Non-Small-Cell Lung	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
Maryland	4
Kansas	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

Last updated: July 28, 2026

Caprelsa (vandetanib) clinical trials update, market analysis, and patent-driven generic/biosimilar outlook

Caprelsa (vandetanib) remains a branded oral targeted therapy for metastatic RET-driven medullary thyroid cancer (MTC) and RET-mutant metastatic non-small cell lung cancer (NSCLC). Patent exclusivity is largely historic and entry risk now tracks around process/formulation patents, label-protected uses, and country-specific patent coverage rather than active regulatory exclusivity. Current commercial momentum depends primarily on RET testing penetration, treatment line dynamics in MTC and NSCLC, and the degree to which newer RET inhibitors displace vandetanib in the prescribing mix.

What is the latest clinical trials landscape for Caprelsa (vandetanib) in RET-driven cancer?

Featured-snippet answer: Public trial activity for vandetanib is concentrated in long-tail updates, combination sequencing, and comparator positioning in RET-mutant or RET-altered disease rather than new pivotal registration studies.

Which indications does the current Caprelsa clinical trial pipeline focus on?

Caprelsa is used in:

• Metastatic medullary thyroid cancer that is not amenable to curative surgery or is metastatic (label geography-dependent).
• Metastatic NSCLC with activating RET mutations (again label geography-dependent).

Contemporary trial activity typically spans:

• RET biomarker-driven enrollment in NSCLC subsets (RET-mutant disease selection).
• Combination or sequencing studies versus other systemic therapies in MTC and NSCLC.
• Real-world evidence protocols and retrospective analyses, which often drive “update” publications more than brand-new interventional registration trials.

What do trial update publications usually measure for vandetanib?

Common endpoints across ongoing or updated analyses include:

• Objective response rate (ORR) and duration of response (DoR).
• Progression-free survival (PFS) by RET status and line of therapy.
• Safety and discontinuation rates for typical vandetanib toxicities (for example QT prolongation, diarrhea, rash).
• Dosing adjustments and dose intensity in routine practice.

How do combination strategies affect the Caprelsa risk-benefit profile in trials?

Where Caprelsa combinations are studied, the clinical issue is usually overlapping toxicities (cardiac and dermatologic) and the ability to maintain therapeutic dose intensity. Trial updates often refine:

• Dose modifications protocols.
• Monitoring intensity (ECG schedules, electrolyte management).
• Patient selection rules based on baseline QTc and comorbidities.

How is Caprelsa (vandetanib) performing commercially, and where does demand come from?

Featured-snippet answer: Caprelsa demand is driven by smaller but persistent RET-biomarker populations, with revenue sensitivity to competing RET inhibitors and broader shifts in treatment guidelines.

What are the demand drivers for Caprelsa in metastatic medullary thyroid cancer?

Key commercial drivers:

• Availability and adoption of RET mutation and RET-driven disease testing.
• Prescriber adherence to RET-targeted therapy in progressive MTC.
• Market share impact from newer RET inhibitors that may move earlier in sequencing.

What are the demand drivers for Caprelsa in RET-mutant metastatic NSCLC?

Key drivers:

• Patient selection quality for activating RET mutations.
• How quickly therapy is started after progression on prior lines.
• Competition against newer-generation RET inhibitors that can be used with different efficacy-safety profiles.

What’s the competitive landscape for vandetanib now?

The competitive set is dominated by next-generation RET inhibitors and broader precision oncology options. This compresses Caprelsa’s addressable market over time through:

• Switching behavior in treatment-naïve or first-line settings (if available in a region’s standard of care).
• Gradual displacement in second-line and beyond where guideline or payer preferences evolve.
• Higher relative use of agents with simpler dosing or better tolerated safety profiles.

When will generic Caprelsa enter, and how do patents affect launch timing by jurisdiction?

Featured-snippet answer: Because Caprelsa is a mature product, generic and authorized generic availability depends on the remaining patent and regulatory exclusivity landscape per country, with timing driven more by patent litigation and country-specific enforcement than by FDA exclusivity milestones.

What patents typically govern vandetanib competition for Caprelsa?

Competition barriers usually include:

• Process patents for manufacturing vandetanib or key intermediates.
• Formulation and polymorph patents (tablet composition, coatings, stability, and manufacturing methods).
• Method-of-use patents tied to specific labeled or clinically supported use patterns.

What Paragraph IV risk exists for Caprelsa?

For a still-marketed small molecule, Paragraph IV risk generally maps to:

• Generic applicants using bioequivalence while attempting to carve around remaining patents.
• Settlement outcomes that can delay generic launches even without full invalidation of the brand estate.

How does “Orange Book status” translate into generic risk?

Practical launch timing risk is usually determined by:

• Whether the listed patents cover the exact drug substance, formulation, or use the generic must match.
• Whether those patents have expired or been found not infringed or invalid.
• Whether a 30-month stay is triggered by a Paragraph IV certification.

What matters most for launch in EU and UK compared with US?

• EU: national patent validity/enforcement through member-state courts and the degree to which patent scope is enforced.
• UK: enforcement through UK courts and local counterpart patents.
• US: FDA Orange Book listings plus district court outcomes for infringement/invalidity.

What is the FDA regulatory status of Caprelsa (vandetanib), and what drives ongoing approvals?

Featured-snippet answer: Caprelsa’s FDA status is that of an established small-molecule oncology drug; current regulatory activity typically supports label maintenance, manufacturing changes, and safety updates rather than fresh exclusivity.

Which FDA document categories usually influence Caprelsa’s commercial availability?

• NDA supplement approvals for manufacturing site or process changes.
• Safety label updates tied to postmarketing experience.
• Changes to dosing guidance based on monitoring requirements.

How does pathway choice influence market outcomes for competing RET drugs?

Market behavior depends on the speed and breadth of new approvals in RET-driven settings. When newer RET drugs gain label coverage for similar patient segments, payers and clinicians tend to shift quickly, reducing the opportunity for late-cycle brand maintenance.

Which formulations and dosing forms of Caprelsa are most likely to be targeted by generics?

Featured-snippet answer: Generic entrants typically seek to match tablet strength(s), release characteristics, and manufacturing processes, while attempting to avoid infringement of formulation-specific patents.

What formulation details usually control generic equivalence?

• Tablet strength and excipient composition.
• Dissolution profile and release kinetics.
• Stability and impurity specifications.

How do capsule/tablet manufacturing patents affect infringement analysis?

• Manufacturing process claims can block “drop-in” generic production even when the API is bioequivalent.
• Formulation claims can restrict copycat excipient and processing details.

How strong is the remaining patent estate for vandetanib (Caprelsa), and what makes it weak or strong?

Featured-snippet answer: For a mature product, the estate strength often shifts from broad drug-substance coverage to narrower process, polymorph, and formulation claims that can still delay entry but are easier to design around than method-of-use claims.

What factors typically strengthen a vandetanib patent estate?

• Broad claims covering intermediates or key synthetic steps.
• Multiple overlapping jurisdictions with enforceable counterparts.
• Tight process parameters where design-arounds are difficult.

What factors typically weaken the estate?

• Expired foundational composition-of-matter coverage.
• Narrow claim scope requiring exact parameter matching.
• Prior art overlap that increases invalidation risk.

What patent litigation and settlements have historically shaped Caprelsa generic entry?

Featured-snippet answer: Vandetanib’s competitive timeline is typically driven by settlement agreements or court outcomes tied to Orange Book-listed patents and the resulting 30-month stay framework.

What to track for litigation-driven timing

• District court decisions on infringement and invalidity.
• ITC investigations, if any, for blocking imports (where relevant).
• Consent judgments or covenants-not-to-sue that effectively define “soft launch” dates.

How does Caprelsa compare with competing RET inhibitors on market share and clinical positioning?

Featured-snippet answer: Caprelsa’s market position is pressured by newer RET inhibitors that often gain earlier preference for tolerability, safety monitoring simplicity, or incremental efficacy in selected subgroups.

What clinical attributes tend to shift prescribing away from vandetanib?

• Differences in QT prolongation burden and monitoring requirements.
• GI toxicity and rash frequency.
• Convenience of dosing and fewer or simpler monitoring requirements.

What commercial levers keep Caprelsa in the mix?

• Existing long-term patient use.
• Payer or formulary preferences in regions where next-generation inhibitors are not fully adopted.
• Availability in specific lines of therapy and reimbursement positioning.

Key market projection logic for Caprelsa (vandetanib) over the next 5 years

Featured-snippet answer: Caprelsa’s trajectory is most sensitive to (1) pace of replacement by newer RET inhibitors, (2) retesting and diagnostic expansion, and (3) regional generic displacement only if remaining patent/process barriers clear.

Scenario model (qualitative, business-facing)

• Base case: Gradual erosion as newer RET inhibitors absorb incremental patients; remaining demand persists in markets with constrained competitive dynamics or where prescribers maintain stable patients.
• Downside: Faster guideline/payer switching plus broader adoption of newer RET inhibitors in first- and second-line settings; sharper volume decline.
• Upside: Slower adoption due to reimbursement limits, tolerability management, or sustained patient continuation in specific regions.

What KPIs should be used to operationalize projections

• Share of prescription starts in RET-mutant NSCLC and RET-driven MTC.
• Time-on-treatment survival proxy in real-world cohorts (continuation rates).
• Formulary tiering changes and net price erosion through tendering and rebates.
• Generic share and tender outcomes where entry is permitted.

Key Takeaways

• Caprelsa (vandetanib) remains a RET-targeted option with demand anchored in metastatic MTC and RET-mutant metastatic NSCLC, but competitive pressure from newer RET inhibitors limits growth.
• Clinical trial “updates” for vandetanib are typically incremental, focused on sequencing, combinations, and safety/monitoring refinements rather than new pivotal registration.
• Generic entry timing is dominated by patent/process/formulation enforcement in each jurisdiction and by litigation or settlement outcomes tied to listed patents.
• Market projections should be modeled around diagnostic testing penetration, guideline/payer switching speed, and regional patent-driven entry rather than relying on new regulatory exclusivity events.

FAQs

1. Does Caprelsa (vandetanib) still have active patent protection in the US for generic entry?
2. What are the most common toxicities that drive dose reduction or discontinuation for vandetanib?
3. How do RET testing rates affect Caprelsa demand in metastatic NSCLC and MTC?
4. What product attributes (API impurity specs, dissolution profile) most influence generic bioequivalence for vandetanib tablets?
5. How do newer-generation RET inhibitors typically change sequencing after progression on vandetanib?

References

1. FDA Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book). U.S. Food and Drug Administration.
2. FDA label and prescribing information for Caprelsa (vandetanib). U.S. Food and Drug Administration.
3. ClinicalTrials.gov. Caprelsa (vandetanib) and vandetanib RET-related studies. National Institutes of Health.