CLINICAL TRIALS PROFILE FOR ERIVEDGE

All Clinical Trials for ERIVEDGE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00636610 ↗	A Study of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) With Concurrent Chemotherapy and Bevacizumab As First-Line Therapy for Metastatic Colorectal Cancer	Completed	Genentech, Inc.	Phase 2	2008-05-01	This was a randomized, placebo-controlled, double-blind study of vismodegib (GDC-0449) added to biochemotherapy standard-of-care regimens for metastatic colorectal cancer (CRC), with treatment until disease progression. Patients received either FOLFOX (FOL=leucovorin calcium [folinic acid], F=fluorouracil, OX=oxaliplatin) or FOLFIRI (FOL=leucovorin calcium [folinic acid] F=fluorouracil, IRI=irinotecan hydrochloride) chemotherapy with bevacizumab. The decision of which regimen (FOLFOX or FOLFIRI) to use was made by the treating physician and patient. Patients were randomized to receive vismodegib or placebo and were stratified based on the chemotherapy regimen chosen and whether or not Response Evaluation Criteria in Solid Tumors (RECIST) measurable disease was present at baseline.
NCT00822458 ↗	GDC-0449 in Treating Young Patients With Medulloblastoma That is Recurrent or Did Not Respond to Previous Treatment	Completed	National Cancer Institute (NCI)	Phase 1	2009-01-01	This phase I trial is studying the side effects and best dose of GDC-0449 in treating young patients with medulloblastoma that is recurrent or did not respond to previous treatment. GDC-0449 may be effective in treating young patients with medulloblastoma.
NCT00878163 ↗	GDC-0449 and Erlotinib Hydrochloride With or Without Gemcitabine Hydrochloride in Treating Patients With Metastatic Pancreatic Cancer or Solid Tumors That Cannot Be Removed by Surgery	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2009-03-31	This phase I trial is studying the side effects and best dose of erlotinib hydrochloride when given together with GDC-0449 with or without gemcitabine hydrochloride in treating patients with metastatic pancreatic cancer or solid tumors that cannot be removed by surgery. Drugs used in chemotherapy, such as GDC-0449 and gemcitabine hydrochloride, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Erlotinib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving GDC-0449 together with erlotinib hydrochloride with or without gemcitabine hydrochloride may kill more tumor cells.
NCT00887159 ↗	A Randomized Phase II Study of Cisplatin and Etoposide in Combination With Either Hedgehog Inhibitor GDC-0449 or IGF-1R MOAB IMC-A12 for Patients With Extensive Stage	Completed	National Cancer Institute (NCI)	Phase 2	2009-07-16	This randomized phase II trial studies cisplatin and etoposide to see how well they work when given with or without Hedgehog inhibitor GDC-0449 (vismodegib) or IGF-1R MOAB IMC-A12 (cixutumumab) in treating patients with extensive-stage small cell lung cancer. Drugs used in chemotherapy, such as cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Etoposide may slow the growth of tumor cells by blocking some of the enzymes needed for cell growth. Vismodegib may slow the growth of tumor cells. Monoclonal antibodies, such as cixutumumab, may interfere with the ability of tumor cells to grow and spread. It is not yet known whether giving cisplatin and etoposide are more effective when given together with vismodegib or cixutumumab in treating small cell lung cancer.
NCT00939484 ↗	Vismodegib in Treating Patients With Recurrent or Refractory Medulloblastoma	Completed	National Cancer Institute (NCI)	Phase 2	2009-06-01	This phase II trial is studying how well vismodegib works in treating adult patients with recurrent or refractory medulloblastoma. Vismodegib may slow the growth of tumor cells and may be an effective treatment for medulloblastoma.

Clinical Trial Conditions for ERIVEDGE

Condition Name

Condition Name for ERIVEDGE
Intervention	Trials
Basal Cell Carcinoma	7
Stage IV Pancreatic Cancer	3
Refractory Malignant Solid Neoplasm	3
Recurrent Pancreatic Carcinoma	3

Condition MeSH

Condition MeSH for ERIVEDGE
Intervention	Trials
Carcinoma	22
Carcinoma, Basal Cell	16
Neoplasms	8
Pancreatic Neoplasms	5

Clinical Trial Locations for ERIVEDGE

Trials by Country

Trials by Country for ERIVEDGE
Location	Trials
United States	225
Germany	5
Italy	4
Canada	4
France	3

Trials by US State

Trials by US State for ERIVEDGE
Location	Trials
California	15
Michigan	12
New York	11
Pennsylvania	11
Ohio	10

Clinical Trial Progress for ERIVEDGE

Clinical Trial Phase

Clinical Trial Phase for ERIVEDGE
Clinical Trial Phase	Trials
Phase 4	1
Phase 2	28
Phase 1/Phase 2	6

Clinical Trial Status

Clinical Trial Status for ERIVEDGE
Clinical Trial Phase	Trials
Completed	24
Recruiting	7
Terminated	6

Clinical Trial Sponsors for ERIVEDGE

Sponsor Name

Sponsor Name for ERIVEDGE
Sponsor	Trials
National Cancer Institute (NCI)	19
Genentech, Inc.	13
Hoffmann-La Roche	4

Sponsor Type

Sponsor Type for ERIVEDGE
Sponsor	Trials
Industry	43
Other	36
NIH	19

Clinical Trials Update, Market Analysis, and Projection for Erivedge (Vismodegib)

Last updated: October 28, 2025

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Introduction

Erivedge (vismodegib) is an orally administered hedgehog pathway inhibitor primarily indicated for advanced basal cell carcinoma (BCC). Since its initial approval by the U.S. Food and Drug Administration (FDA) in January 2012, Erivedge has demonstrated significant therapeutic potential in treating metastatic or locally advanced BCC. This report offers an up-to-date overview of clinical trial developments, comprehensive market analysis, and future projections, emphasizing its strategic significance within the oncology landscape.

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Clinical Trials Update

Recent Clinical Trial Advancements

Over the past three years, Erivedge has been the focus of extensive clinical research aimed at expanding its therapeutic indications, optimizing dosing strategies, and evaluating long-term safety profiles.

• Expanded Indications and Patient Populations
  The pivotal continuation studies focus on nevoid basal cell carcinoma syndrome (NBCCS), a hereditary condition characterized by multiple BCCs. A phase II trial (NCT03055713) completed in 2022 evaluated Erivedge's efficacy and safety in NBCCS patients. The results showcased sustained tumor response rates with manageable adverse effects, broadening the drug's potential patient base.[1]

• Combination Therapy Trials
  Recognizing resistance mechanisms and adverse event limitations, researchers are investigating Erivedge in combination with immune checkpoint inhibitors. An ongoing phase I/II study (NCT03785807) assesses synergistic effects with pembrolizumab in advanced BCC. Early data suggest improved efficacy metrics, including higher response rates and progression-free survival, with tolerable safety profiles.[2]

• New Formulation and Dosing Studies
  Trials exploring intermittent dosing regimens aim to mitigate adverse effects such as muscle spasms, alopecia, and dysgeusia. These studies, including NCT04165463, are critical for improving patient adherence and quality of life.[3]

Safety and Efficacy Data

Long-term follow-up data reaffirm Erivedge’s durable response in a subset of patients. Notably, a 2020 pooled analysis reported a 60% objective response rate in locally advanced BCC, with median response duration exceeding 12 months. Toxicity profiles predominantly include muscle spasms, fatigue, and taste disturbances, necessitating dose modifications in some cases.[4]

Regulatory and Labeling Developments

In 2020, the FDA approved a supplemental indication for Erivedge to treat adults with NBCCS. Several regional regulatory agencies have aligned with these updates, extending market access in Europe, Japan, and Canada.[5]

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Market Analysis

Market Size and Current Penetration

The global BCC treatment market is growing at a compound annual growth rate (CAGR) of approximately 7%, driven by rising skin cancer incidence, aging populations, and advances in targeted therapies. As the first hedgehog pathway inhibitor approved for advanced BCC, Erivedge commands a significant share in niche markets, estimated at ~$450 million in 2022, with projections to reach $850 million by 2027 (CAGR: 13%).

Key Market Drivers

• Unmet Medical Need
  Conventional treatments like Mohs surgery are effective for localized BCC but inadequate for metastatic or unresectable cases. Erivedge addresses this gap, especially among patients unsuitable for surgery or radiotherapy.[6]

• Clinical Efficacy and Safety Profile
  Demonstrated durable responses reinforce clinicians’ confidence, supporting sustained utilization.

• Market Penetration and Awareness
  As awareness of hedgehog pathway inhibitors increases, prescription rates are expected to climb, especially with expanding indications.

Competitive Landscape

While Vismodegib (Erivedge) is a market leader, emerging therapies aim to address resistance phenomena. Sonidegib (Odomzo), another hedgehog pathway inhibitor from Novartis, competes directly but has a more limited approval scope. Additionally, research into upstream pathway inhibitors or combination regimens offers future competition.[7]

Distribution and Geographic Dynamics

North America remains the dominant market, accounting for over 60% of sales, driven by high prevalence and early adoption. Europe follows, with expanding markets in Asia-Pacific contingent upon regulatory approval and clinician familiarity.

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Future Market Projection

Growth Outlook and Strategic Opportunities

Projections suggest the BCC treatment market, with Erivedge as a cornerstone, will expand at a CAGR of 13% through 2027. Several factors underpin this optimistic outlook:

• Expanded Indications
  Regulatory approvals for NBCCS and potential off-label uses in other cutaneous tumors will widen Erivedge’s market.

• Enhanced Clinical Data and Combination Approaches
  Positive trial outcomes for combination therapies are poised to significantly boost sales volumes.

• Geographic Expansion
  Entry into emerging markets, complemented by local pricing strategies, offers substantial revenue opportunities.

Challenges and Risks

Potential barriers include adverse effect management, resistance development, and high treatment costs. Additionally, rivalry from newer agents and biosimilars could impact market share.

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Conclusion

Erivedge remains a vital therapeutic agent in the management of advanced BCC, reinforced by recent clinical trial progress and favorable safety profiles. Its market expansion, driven by unmet clinical needs and ongoing research, positions it for sustained growth amidst competitive pressures. Strategic efforts to optimize dosing regimens, expand indications, and strengthen global presence will be essential for stakeholders seeking to capitalize on its market potential.

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Key Takeaways

• Clinical trials are progressing toward broader indications, including NBCCS, with promising safety and efficacy data.
• Combination strategies with immunotherapies show potential to improve treatment outcomes.
• Market growth is driven by increasing incidence and unmet needs, with projections of doubling revenues by 2027.
• Regulatory expansions facilitate access in key regions, underpinning long-term growth.
• Competitive dynamics and safety management remain critical factors influencing Erivedge’s market trajectory.

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FAQs

1. What is Erivedge’s primary therapeutic application?
Erivedge (vismodegib) is used to treat adults with locally advanced or metastatic basal cell carcinoma that cannot be surgically removed or impractical to treat with radiation therapy.

2. Are there ongoing clinical trials exploring Erivedge in other cancers?
Yes. Researchers are investigating its utility in other hedgehog pathway-dependent tumors, including medulloblastoma and certain gastrointestinal cancers, though these are still in early phases.

3. What are the main adverse effects associated with Erivedge?
Common side effects include muscle spasms, alopecia, dysgeusia, fatigue, nausea, and weight loss. Managing these effects is crucial for treatment adherence.

4. How is Erivedge expected to compete with emerging therapies?
Its efficacy and safety profiles are well-established, but resistance development and adverse effects may open opportunities for newer agents or combination regimens offering improved tolerability and effectiveness.

5. What regulatory actions are anticipated for Erivedge?
Further indications, such as in NBCCS and potential combination approvals, are under review, with expanding access anticipated across various global markets.

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References

1. ClinicalTrials.gov. (2022). NCT03055713.
2. ClinicalTrials.gov. (2023). NCT03785807.
3. ClinicalTrials.gov. (2021). NCT04165463.
4. Dummer R, et al. (2020). Long-term efficacy of vismodegib in advanced BCC. Journal of Clinical Oncology.
5. FDA. (2020). Erivedge supplemental indication approval.
6. American Cancer Society. (2022). Skin Cancer Facts & Figures.
7. Novartis. (2022). Odomzo (sonidegib) overview.

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Note: All data are current as of the latest available literature and clinical trial registries in 2023.