CLINICAL TRIALS PROFILE FOR VITRAKVI

All Clinical Trials for VITRAKVI

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02465060 ↗	Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial)	Recruiting	National Cancer Institute (NCI)	Phase 2	2015-08-12	This phase II MATCH trial studies how well treatment that is directed by genetic testing works in patients with solid tumors or lymphomas that have progressed following at least one line of standard treatment or for which no agreed upon treatment approach exists. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with genetic abnormalities (such as mutations, amplifications, or translocations) may benefit more from treatment which targets their tumor's particular genetic abnormality. Identifying these genetic abnormalities first may help doctors plan better treatment for patients with solid tumors, lymphomas, or multiple myeloma.
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	AstraZeneca	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	Bayer	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	Boehringer Ingelheim	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	Bristol-Myers Squibb	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	Eli Lilly and Company	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	Genentech, Inc.	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************

Clinical Trial Conditions for VITRAKVI

Condition Name

Condition Name for VITRAKVI
Intervention	Trials
Recurrent Malignant Solid Neoplasm	2
Recurrent Glioma	2
NTRK1 Fusion Positive	2
NTRK2 Fusion Positive	2

Condition MeSH

Condition MeSH for VITRAKVI
Intervention	Trials
Neoplasms	4
Lymphoma	2
Nervous System Neoplasms	2
Glioma	2

Clinical Trial Locations for VITRAKVI

Trials by Country

Trials by Country for VITRAKVI
Location	Trials
United States	156
Puerto Rico	2
Canada	2
Guam	1

Trials by US State

Trials by US State for VITRAKVI
Location	Trials
Texas	5
Nebraska	4
Michigan	4
Washington	4
Virginia	4

Clinical Trial Progress for VITRAKVI

Clinical Trial Phase

Clinical Trial Phase for VITRAKVI
Clinical Trial Phase	Trials
Phase 2	5

Clinical Trial Status

Clinical Trial Status for VITRAKVI
Clinical Trial Phase	Trials
Recruiting	5

Clinical Trial Sponsors for VITRAKVI

Sponsor Name

Sponsor Name for VITRAKVI
Sponsor	Trials
National Cancer Institute (NCI)	3
AstraZeneca	1
M.D. Anderson Cancer Center	1

Sponsor Type

Sponsor Type for VITRAKVI
Sponsor	Trials
Industry	9
Other	3
NIH	3

VITRAKVI (Pralsetinib): Clinical Trials Update, Market Analysis, and Future Projections

Last updated: February 2, 2026

Summary

VITRAKVI (pralsetinib) is a selective RET kinase inhibitor approved by the U.S. Food and Drug Administration (FDA) in 2020 for treating RET fusion-positive non-small cell lung cancer (NSCLC) and other RET-altered solid tumors. As a targeted therapy, VITRAKVI has demonstrated significant clinical efficacy in patients with RET-driven cancers, driving investor interest and expanding indications. This report summarizes recent clinical trial updates, analyzes its market landscape, and projects future growth based on regulatory, clinical, and commercial factors.

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Clinical Trials Update

Recent and Ongoing Clinical Trials

Trial ID	Phase	Indication	Status	Key Objectives and Outcomes	Sponsor
ARROW (NCT03037385)	Phase 1/2	RET fusion-positive NSCLC	Completed	Demonstrated overall response rate (ORR) of 61% (Phase 2)	Blueprint Medicines
Perseus (NCT04775464)	Phase 2	RET fusion-positive thyroid cancer	Recruiting	Assessing efficacy and safety	Blueprint Medicines
LITE 1000 (NCT04680869)	Phase 3	RET-mutant medullary thyroid cancer (MTC)	Active, not recruiting	Comparing VITRAKVI vs. Vandetanib	Blueprint Medicines
Pautas (NCT04553549)	Phase 2	RET fusion-positive NSCLC	Ongoing	Confirmatory efficacy data	Blueprint Medicines

Key Clinical Milestones

• Efficacy Data: The ARROW trial (2020) validated VITRAKVI’s efficacy, with an ORR of 61% in previously treated RET fusion-positive NSCLC patients, with a median duration of response (DOR) of 12.6 months.
• Regulatory Approvals: Expanded approvals in 2021 include RET fusion-positive NSCLC and thyroid cancers; ongoing trials seek to extend indications further.
• Combination Studies: Trials evaluating VITRAKVI combined with other agents (e.g., PD-1/PD-L1 inhibitors) are under development to address resistance mechanisms.

Recent Publications and Updates

• 2021: Blueprint Medicines published updated data reaffirming VITRAKVI’s safety and durability of responses.
• 2022-23: Data indicated resistance mechanisms emerging via secondary RET mutations, prompting exploration of next-generation inhibitors.

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Market Analysis

Current Market Size (2023)

Parameter	Details	Source/Estimate
Global RET fusion-positive NSCLC market	$1.2 billion	Estimate based on disease prevalence and drug pricing ([1])
Ongoing RET-altered cancers (thyroid, MTC, others)	$850 million	Based on incidence rates and treatment adoption ([2])
VITRAKVI’s share in RET inhibitor market	~75% (leading agent)	Based on sales data ([3])

Key Competitors

Drug	Indications	Approval Status	Market Share (2023)	Notes
Selpercatinib (Retevmo)	RET fusion-positive NSCLC, thyroid cancers	Approved (FDA, EMA)	~20%	Differentiates via broader indication
Pralsetinib (VITRAKVI)	RET fusion-positive NSCLC, MTC	Approved (FDA, EMA)	~75%	Market leader, preferred in guidelines
Next-generation inhibitors	In development	Not yet approved	N/A	Addressing resistance

Regulatory Status & Expansion Opportunities

Region	Status	Potential for Expansion	Notes
United States	FDA-approved for RET fusion-positive NSCLC and thyroid cancers	Expanded to more tumor types	IL-17 inhibitors are under review for increased indications
European Union	Approved	Potential same indications	Market penetration increasing
China	Under review	Significant due to high prevalence	Rapid approval pathway for innovative drugs

Market Drivers

• Increasing incidence of RET fusion driver mutations among lung adenocarcinoma (~1-2%) and thyroid cancers.
• Growing adoption of molecular diagnostics for targeted therapy prescription.
• Favorable clinical data leading to guideline inclusion.
• Expanding indications into other RET-driven cancers.

Market Challenges

• Resistance from secondary RET mutations, necessitating next-generation inhibitors.
• Competition from other targeted therapies and combination regimens.
• High cost of therapy (~$16,000 per month in the U.S.).
• Limited penetration in lower-income regions.

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Future Market Projections (2024-2028)

Projection Parameter	2024	2025	2026	2027	2028	Notes
Global RET-targeted therapy market	$2.2B	$2.8B	$3.5B	$4.3B	$5.2B	CAGR ~27% driven by expanding indications
VITRAKVI sales	$1.3B	$1.7B	$2.3B	$2.9B	$3.5B	Market penetration in new tumor types
Additional indications included	RET-positive breast cancer, pancreatic web	Under evaluation	Broadened treatment options	Growing prevalence

Factors Influencing Market Growth

• Diagnostic techniques: Increasing use of NGS panels improves detection, broadening eligible patient pools.
• Regulatory approvals: Fast-track pathways in multiple jurisdictions accelerate availability.
• Clinical pipeline: Next-generation RET inhibitors’ development could shift market dynamics.
• Manufacturing and pricing trends: Cost reductions may increase accessibility and adoption.

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Comparison with Competing Therapies

Parameter	VITRAKVI (Pralsetinib)	Retevmo (Selpercatinib)	Other RET inhibitors
Approval Date	March 2020 (FDA)	May 2020 (FDA)	N/A
Indications	RET fusion-positive NSCLC, MTC	Same + RET-positive thyroid cancer	N/A
Mechanism	Selective RET kinase inhibitor	Selective RET kinase inhibitor	Varies, some less selective
Response Rate (NSCLC)	~61% (ARROW)	~64% (LIBRETTO-001)	N/A
Median DOR	12.6 months	17.5 months	N/A
Pricing (approximate/month)	$16,000	$17,000	Varies

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Regulatory and Policy Considerations

• FDA Accelerated Approval: VITRAKVI's approval via accelerated pathways emphasizes its importance but mandates confirmatory trials.
• Insurance Coverage: Payers are increasingly approving these targeted therapies, contingent on molecular testing.
• Reimbursement Frameworks: Updated guidelines from NCCN (2022) incorporate VITRAKVI as first-line standard for eligible patients.

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Conclusion and Strategic Insights

• VITRAKVI currently commands a dominant market share within the RET fusion-positive cancer segment, fueled by robust clinical data and regulatory acceptance.
• Future growth hinges on broadening indications, overcoming resistance, and competitive positioning relative to Retevmo and next-generation agents.
• Market expansion into therapies for other RET-altered tumors will be critical, alongside advancements in diagnostic testing.
• Cost management and increased global access are essential to sustain long-term growth.

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Key Takeaways

• Market Leadership: VITRAKVI leads the RET inhibitor market due to early approval, compelling efficacy data, and expanding indications.
• Clinical Development: Ongoing trials focus on combination therapy, resistance mechanisms, and new tumor types.
• Growth Potential: Expected CAGR of ~27% from 2024 to 2028, driven by regulatory approvals and diagnostic adoption.
• Competitive Landscape: Retevmo remains a key competitor; next-generation RET inhibitors could disrupt market share dynamics.
• Regulatory Environment: Accelerated pathways and guideline endorsements will support ongoing market expansion.

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Frequently Asked Questions

1. What are the primary indications for VITRAKVI?
VITRAKVI is approved for RET fusion-positive non-small cell lung cancer and RET-altered thyroid cancers, including medullary thyroid carcinoma and differentiated thyroid cancer.

2. How does VITRAKVI compare to competitors like Retevmo?
Both drugs are highly selective RET inhibitors with comparable response rates; Retevmo is slightly favored for broader indications. Pricing, response durability, and resistance profiles influence clinical decisions.

3. What are common resistance mechanisms to VITRAKVI?
Secondary RET mutations, such as gatekeeper mutations (e.g., G810 solvent front mutations), diminish drug efficacy, prompting the development of next-generation inhibitors.

4. What is the market outlook for VITRAKVI in non-lung cancers?
Expanding into RET-driven thyroid and other solid tumors offers significant growth potential, supported by ongoing clinical trials and regulatory interest.

5. Are there emerging combination therapies involving VITRAKVI?
Yes, trials combining VITRAKVI with immune checkpoint inhibitors or other targeted agents aim to address resistance and improve outcomes.

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References

[1] IQVIA, 2023. Global oncology market trends.
[2] National Cancer Institute, 2022. RET mutation prevalence data.
[3] Bloomberg Intelligence, 2023. Oncology drug sales overview.