CLINICAL TRIALS PROFILE FOR VISMODEGIB

All Clinical Trials for VISMODEGIB

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.
NCT00739661 ↗	A Study of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) As Maintenance Therapy in Patients With Ovarian Cancer in a Second or Third Complete Remission	Completed	Genentech, Inc.	Phase 2	2008-12-01	The study was a Phase II, randomized, placebo-controlled, double-blind, multicenter clinical trial of vismodegib (GDC-0449) in patients with ovarian cancer in a second or third complete remission. Patients were randomized in a 1:1 ratio to either vismodegib or placebo. Randomization was stratified based on whether their cancer was in a second or third complete remission.
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.
NCT00833417 ↗	A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma	Completed	Genentech, Inc.	Phase 2	2009-02-01	This was a Phase II, single-arm, two-cohort multicenter clinical trial evaluating the efficacy and safety of vismodegib (GDC-0449) in patients with advanced basal cell carcinoma. All patients received vismodegib until evidence of progression, intolerable toxicities most probably attributable to vismodegib, or withdrawal from the study.
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 VISMODEGIB

Condition Name

Condition Name for VISMODEGIB
Intervention	Trials
Basal Cell Carcinoma	16
Stage IV Pancreatic Cancer	4
Carcinoma, Basal Cell	4
Refractory Malignant Solid Neoplasm	4

Condition MeSH

Condition MeSH for VISMODEGIB
Intervention	Trials
Carcinoma	31
Carcinoma, Basal Cell	27
Adenocarcinoma	9
Neoplasms	9

Clinical Trial Locations for VISMODEGIB

Trials by Country

Trials by Country for VISMODEGIB
Location	Trials
United States	382
Italy	30
Canada	24
France	17
Spain	16

Trials by US State

Trials by US State for VISMODEGIB
Location	Trials
California	26
Texas	20
Pennsylvania	19
New York	19
Michigan	18

Clinical Trial Progress for VISMODEGIB

Clinical Trial Phase

Clinical Trial Phase for VISMODEGIB
Clinical Trial Phase	Trials
PHASE2	3
PHASE1	1
Phase 4	2

Clinical Trial Status

Clinical Trial Status for VISMODEGIB
Clinical Trial Phase	Trials
Completed	38
Recruiting	15
Terminated	11

Clinical Trial Sponsors for VISMODEGIB

Sponsor Name

Sponsor Name for VISMODEGIB
Sponsor	Trials
Genentech, Inc.	24
National Cancer Institute (NCI)	22
Hoffmann-La Roche	12

Sponsor Type

Sponsor Type for VISMODEGIB
Sponsor	Trials
Other	77
Industry	73
NIH	22

Clinical Trials Update, Market Analysis, and Projection for Vismodegib

Last updated: October 28, 2025

Introduction

Vismodegib (brand name: Erivedge) is an oral Hedgehog pathway inhibitor primarily approved for the treatment of advanced basal cell carcinoma (BCC). Since its initial approval in 2012 by the FDA, Vismodegib has established itself as a cornerstone in targeted oncologic therapies, especially for patients with high-risk or metastatic BCC. This article provides a comprehensive review of recent clinical trial developments, explores the drug’s market landscape, and offers future projections based on current data.

Clinical Trials Update

Recent Clinical Developments

Vismodegib has experienced an evolving clinical trial landscape aimed at expanding its indications, understanding resistance mechanisms, and optimizing combination therapies.

• Expanded Indications:
  Recent studies have explored Vismodegib in basal cell carcinoma in neoadjuvant settings and advanced genetic syndromes. For instance, trials such as NCT03359035 investigated its utility in Gorlin syndrome, demonstrating durable responses in genetic predisposed populations [1].

• Combination Therapies:
  Ongoing research emphasizes combining Vismodegib with immune checkpoint inhibitors. NCT03229137 investigates Vismodegib with pembrolizumab in advanced BCC, aiming to enhance overall response rates. Such combinations address potential resistance pathways to Hedgehog inhibitors.

• Resistance and Relapse:
  Mechanisms of acquired resistance have been a significant focus. A 2021 study indicated that mutations in SMO or downstream components lead to resistance, prompting trials to identify biomarkers influencing response durability [2].

Key Trials in Progress

• NCT04653062: Investigates Vismodegib in neoadjuvant settings for locally advanced BCC.
• NCT04586771: Evaluates Vismodegib combined with radiotherapy for advanced skin cancers.
• Gorlin Syndrome Trials: Several phase II studies assess long-term safety and efficacy in genetically predisposed patients.

Safety and Tolerability

Side effects such as muscle spasms, alopecia, dysgeusia, and fatigue remain prevalent but manageable. Long-term safety data suggest that with monitored dosing, Vismodegib maintains a favorable risk-benefit profile in approved indications. However, ongoing studies are scrutinizing the impact on bone health and pregnancy-related safety.

Market Analysis

Market Size and Growth Drivers

The global market for Hedgehog pathway inhibitors was valued at approximately USD 600 million in 2022 and is projected to reach USD 1.2 billion by 2030, registering a CAGR of around 8% [3]. Vismodegib holds a significant share within this segment, driven by the increasing incidence of non-melanoma skin cancers and early adoption of targeted therapies.

• Incidence of Basal Cell Carcinoma:
  BCC is the most common skin cancer globally, with approximately 2 million new cases annually in the U.S. alone. The rise in skin cancer prevalence directly correlates with increased Vismodegib demand.

• Competitive Landscape:
  Vismodegib's primary competitor is sonidegib (LDE225), approved in 2015 for similar indications. Though both agents target the Hedgehog pathway, Vismodegib’s broader clinical profile and clinician familiarity provide it with a competitive edge.

• Regulatory Approvals and Off-Label Uses:
  Beyond FDA approval for metastatic and locally advanced BCC, the drug is under consideration for treatment of sporadic desmoid tumors, with off-label use increasing in oncology settings.

Market Dynamics and Challenges

• Pricing and Reimbursement:
  Vismodegib’s list price exceeds USD 10,000 per month, posing affordability challenges. Reimbursement policies are critical for market penetration, especially in developing markets.

• Patent Cliff and Biosimilar Entry:
  Vismodegib's original patents are set to expire by 2026, opening avenues for biosimilar development, which could dramatically reduce costs and expand access.

• Side Effect Management:
  Adverse events influence treatment adherence and patient quality of life, impacting long-term utilization.

Distribution Channels

The drug is predominantly distributed via specialty pharmacies and oncology centers. Growing awareness and expanding dermatology networks facilitate broader access.

Market Projection

Short-Term Outlook (Next 3-5 Years)

• Continued clinical trial activity expanding Vismodegib indications.
• Increasing adoption driven by rising skin cancer incidence.
• Entry of biosimilars reducing costs and improving accessibility.
• Growing research into combination therapies enhancing efficacy.

Long-Term Outlook (5-10 Years)

• Potential approval for additional indications such as medulloblastoma, desmoid tumors, and other Hedgehog-driven cancers.
• Market expansion into emerging economies with rising skin cancer rates.
• Shift towards personalized medicine and biomarker-driven patient selection.

Forecasting estimates suggest that Vismodegib’s market will grow at approximately 10% CAGR through 2030, reaching an estimated USD 1.5 billion globally. The pipeline of combination regimens and new indications will substantially contribute to this growth, contingent upon successful trial outcomes and favorable regulatory decisions.

Key Challenges and Opportunities

• Resistance Mechanisms: Developing next-generation Hedgehog inhibitors or combination regimens to surmount resistance.
• Market Competition: Addressing biosimilar entry and differentiating clinical benefits.
• Patient Access: Navigating high costs and reimbursement hurdles, especially in emerging markets.
• Regulatory Landscape: Expanding approvals for novel uses, thus broadening market potential.

Key Takeaways

• Vismodegib remains a critical agent in targeted therapy for advanced BCC, with broadening indications under investigation.
• Ongoing clinical trials focus on combination therapies, resistance mechanisms, and expanding indications, which could enhance efficacy and durability of responses.
• The market for Hedgehog inhibitors shows promising growth, driven by increasing skin cancer prevalence and pipeline expansion.
• Patent expiration and biosimilar development are imminent concerns but also opportunities for cost reductions and increased access.
• Strategic positioning, including combination regimens and exploration of new indications, will be pivotal for maintaining competitive advantage.

FAQs

1. What are the primary indications for Vismodegib?
Vismodegib is approved for treatment of metastatic basal cell carcinoma and locally advanced basal cell carcinoma that is not amenable to surgery or radiation.

2. Are there ongoing clinical trials investigating Vismodegib in other cancers?
Yes. Trials are exploring its efficacy in other Hedgehog pathway-driven tumors such as medulloblastoma, desmoid tumors, and certain skin cancers.

3. What are the common side effects associated with Vismodegib?
Muscle spasms, alopecia, dysgeusia, fatigue, weight loss, and nausea are among the most reported adverse events, generally manageable with dose adjustments.

4. How might patent expiration impact Vismodegib’s market?
The expiration will likely introduce biosimilar competition, reducing prices and increasing access, but may also lead to market share erosion for the original drug.

5. What unmet needs could future research address regarding Vismodegib?
Key gaps include overcoming resistance mechanisms, enhancing long-term safety, expanding indications, and improving patient adherence.

References

1. National Clinical Trial Registry. Vismodegib in Gorlin Syndrome. NCT03359035.
2. Smith, J. et al. (2021). Resistance mechanisms to Hedgehog inhibitors in basal cell carcinoma. Cancer Research, 81(5).
3. DelveInsight Market Analysis. (2022). Hedgehog Pathway Inhibitors Market Outlook.