CLINICAL TRIALS PROFILE FOR TEMOZOLOMIDE

505(b)(2) Clinical Trials for temozolomide

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT00876993 ↗	Study of Irinotecan and Bevacizumab With Temozolomide in Refractory/Relapsed Central Nervous System (CNS) Tumors	Completed	Brain Tumor Alliance	Phase 1	2008-09-01	Bevacizumab, irinotecan, and temozolomide are three agents shown to have promising activity in a variety of central nervous system tumors. No prospective studies have been published or are currently in progress within the major consortiums with this combination of drugs. Brain tumors are the second most common cause of cancer in pediatrics and the leading cause of cancer death in children. For children with High Grade Gliomas or with relapsed/refractory brain tumors, new agents in new combinations are needed. Historical data shows that newly diagnosed high grade gliomas 5 year progression free survival is 28-42%. Recurrent malignant gliomas median survival is 3-9 months. Recurrent medulloblastoma's 2 years survival is 9%. This study is a phase I study designed to provide an objective observation of toxicity and establish a maximum tolerated dose of this combination. In addition, this study will observe the response of children with relapsed or refractory central nervous system tumors.
New Combination	NCT00876993 ↗	Study of Irinotecan and Bevacizumab With Temozolomide in Refractory/Relapsed Central Nervous System (CNS) Tumors	Completed	The V Foundation	Phase 1	2008-09-01	Bevacizumab, irinotecan, and temozolomide are three agents shown to have promising activity in a variety of central nervous system tumors. No prospective studies have been published or are currently in progress within the major consortiums with this combination of drugs. Brain tumors are the second most common cause of cancer in pediatrics and the leading cause of cancer death in children. For children with High Grade Gliomas or with relapsed/refractory brain tumors, new agents in new combinations are needed. Historical data shows that newly diagnosed high grade gliomas 5 year progression free survival is 28-42%. Recurrent malignant gliomas median survival is 3-9 months. Recurrent medulloblastoma's 2 years survival is 9%. This study is a phase I study designed to provide an objective observation of toxicity and establish a maximum tolerated dose of this combination. In addition, this study will observe the response of children with relapsed or refractory central nervous system tumors.
New Combination	NCT00876993 ↗	Study of Irinotecan and Bevacizumab With Temozolomide in Refractory/Relapsed Central Nervous System (CNS) Tumors	Completed	V Foundation	Phase 1	2008-09-01	Bevacizumab, irinotecan, and temozolomide are three agents shown to have promising activity in a variety of central nervous system tumors. No prospective studies have been published or are currently in progress within the major consortiums with this combination of drugs. Brain tumors are the second most common cause of cancer in pediatrics and the leading cause of cancer death in children. For children with High Grade Gliomas or with relapsed/refractory brain tumors, new agents in new combinations are needed. Historical data shows that newly diagnosed high grade gliomas 5 year progression free survival is 28-42%. Recurrent malignant gliomas median survival is 3-9 months. Recurrent medulloblastoma's 2 years survival is 9%. This study is a phase I study designed to provide an objective observation of toxicity and establish a maximum tolerated dose of this combination. In addition, this study will observe the response of children with relapsed or refractory central nervous system tumors.
New Combination	NCT00876993 ↗	Study of Irinotecan and Bevacizumab With Temozolomide in Refractory/Relapsed Central Nervous System (CNS) Tumors	Completed	Johns Hopkins All Children's Hospital	Phase 1	2008-09-01	Bevacizumab, irinotecan, and temozolomide are three agents shown to have promising activity in a variety of central nervous system tumors. No prospective studies have been published or are currently in progress within the major consortiums with this combination of drugs. Brain tumors are the second most common cause of cancer in pediatrics and the leading cause of cancer death in children. For children with High Grade Gliomas or with relapsed/refractory brain tumors, new agents in new combinations are needed. Historical data shows that newly diagnosed high grade gliomas 5 year progression free survival is 28-42%. Recurrent malignant gliomas median survival is 3-9 months. Recurrent medulloblastoma's 2 years survival is 9%. This study is a phase I study designed to provide an objective observation of toxicity and establish a maximum tolerated dose of this combination. In addition, this study will observe the response of children with relapsed or refractory central nervous system tumors.

All Clinical Trials for temozolomide

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00003062 ↗	Temozolomide in Patients With Progressive or Recurrent Non-small Cell Lung Cancer	Completed	European Organisation for Research and Treatment of Cancer - EORTC	Phase 2	1997-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effect of temozolomide in patients with progressive or recurrent stage IV non-small cell lung cancer, with or without brain metastases, who have not been treated for metastatic disease with chemotherapy.
NCT00003176 ↗	Temozolomide and Carmustine in Treating Patients With Anaplastic Glioma	Completed	National Cancer Institute (NCI)	Phase 2	1998-03-25	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of temozolomide and carmustine in treating patients with anaplastic glioma.
NCT00003176 ↗	Temozolomide and Carmustine in Treating Patients With Anaplastic Glioma	Completed	North American Brain Tumor Consortium	Phase 2	1998-03-25	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of temozolomide and carmustine in treating patients with anaplastic glioma.
NCT00003176 ↗	Temozolomide and Carmustine in Treating Patients With Anaplastic Glioma	Completed	Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	Phase 2	1998-03-25	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of temozolomide and carmustine in treating patients with anaplastic glioma.

Clinical Trial Conditions for temozolomide

Condition Name

Condition Name for temozolomide
Intervention	Trials
Glioblastoma	240
Glioblastoma Multiforme	138
Brain and Central Nervous System Tumors	89
Glioma	59

Condition MeSH

Condition MeSH for temozolomide
Intervention	Trials
Glioblastoma	511
Glioma	214
Central Nervous System Neoplasms	114
Nervous System Neoplasms	109

Clinical Trial Locations for temozolomide

Trials by Country

Trials by Country for temozolomide
Location	Trials
Japan	81
Netherlands	62
Switzerland	58
Belgium	56
Austria	36

Trials by US State

Trials by US State for temozolomide
Location	Trials
California	214
Texas	189
New York	180
North Carolina	163
Pennsylvania	161

Clinical Trial Progress for temozolomide

Clinical Trial Phase

Clinical Trial Phase for temozolomide
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	16
PHASE2	45

Clinical Trial Status

Clinical Trial Status for temozolomide
Clinical Trial Phase	Trials
Completed	407
Recruiting	241
Not yet recruiting	113

Clinical Trial Sponsors for temozolomide

Sponsor Name

Sponsor Name for temozolomide
Sponsor	Trials
National Cancer Institute (NCI)	242
Schering-Plough	37
Merck Sharp & Dohme Corp.	36

Sponsor Type

Sponsor Type for temozolomide
Sponsor	Trials
Other	1338
Industry	543
NIH	255

Clinical Trials Update, Market Analysis, and Projection for Temozolomide

Last updated: January 26, 2026

Executive Summary

Temozolomide (TMZ), an oral alkylating agent approved primarily for the treatment of glioblastoma multiforme (GBM) and certain types of melanoma, remains a cornerstone in neuro-oncology therapy. Recent advancements focus on expanding its indications, enhancing its efficacy, and improving patient outcomes through combination therapies and novel formulations. The global market for Temozolomide is experiencing moderate growth driven by rising incidence of glioblastoma, increased clinical research activity, and strategic collaborations. Projected compounded annual growth rate (CAGR) over the next five years is approximately 3.5-4%, with potential acceleration tied to emerging indications and combination regimens.

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Clinical Trials Update

Current Landscape and Recent Developments

Parameter	Details
Total Active Trials (as of Q1 2023)	40+ (Source: ClinicalTrials.gov)
Indications Under Investigation	Glioblastoma, melanoma, various brain tumors, refractory cancers, combinations with immunotherapies (e.g., PD-1 inhibitors)
Phase Distribution	Phase I (10%), Phase II (55%), Phase III (15%),others (20%)
Key Trials	- NCT04567920: Phase III evaluating TMZ + Tumor Treating Fields (TTFields) in GBM. - NCT03889481: Phase II assessing TMZ in recurrent glioma. - NCT03545374: Evaluating combination of TMZ with pembrolizumab in melanoma.

Impact of Recent Trials

• Combination Therapies: Numerous studies focus on combining TMZ with immunotherapies, targeted agents, and radiation to improve survival metrics.
• Expanded Indications: Trials are exploring efficacy in pediatric brain tumors and metastatic cancers outside CNS.
• Biomarker Research: Emerging studies evaluate MGMT methylation status as a predictor of TMZ responsiveness, guiding personalized medicine.

Regulatory and Approval Status

• Approved Uses: EMEA and FDA approve TMZ for newly diagnosed and recurrent GBM, and melanoma.
• Emerging Approvals: Trials for other indications, such as anaplastic astrocytoma, are progressing with pivotal data expected by 2024.

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Market Analysis

Market Size and Growth Drivers

Parameter	2022 Estimates	Notes
Global Market Size	USD 600 million	(Source: Grand View Research, 2022)
Regional Breakdown	North America (55%), Europe (25%), Asia-Pacific (15%), ROW (5%)	North America dominates due to high glioblastoma prevalence and advanced healthcare infrastructure.
Market CAGR (2023–2028)	3.5-4%	Driven by rising patient prevalence, ongoing clinical trials, and pipeline expansion.

Key Market Players

Company	Product Name	Market Share (Approx.)	Additional Notes
Merck KGaA / AstraZeneca	Temodal® (brand for TMZ)	~70%	Leading provider, with licensed patent expirations in select markets.
Mylan (now part of Viatris)	Generic Temozolomide	20%	Increasing market share post-patent expiry.
Others	Various generics	10%	Emerging players in Asia and Europe.

Pricing and Reimbursement

• Pricing Range: USD 50–150 per capsule depending on brand and dosage.
• Reimbursement Policies: Coverage varies; higher in mature markets like the US and Europe, limited in emerging regions.

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Market Projection (2023-2028)

Year	Projected Market Size (USD)	Growth Rate	Drivers	Risks
2023	USD 625 million		Continued clinical trials, new indications	Patent cliffs, generic competition
2024	USD 650 million	4%	Expanded indications, pipeline progress	Regulatory hurdles
2025	USD 680 million	4.6%	Integration of combination therapies	Market entry barriers
2026	USD 710 million	4.4%	Adoption of personalized treatment approaches	Pricing pressures
2027	USD 740 million	4.2%	Global expansion	Patent expiries in emerging markets
2028	USD 770 million	4%	Increased use in combination regimens	Competitive innovations

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Comparison: Temozolomide vs. Similar Agents

Parameter	Temozolomide	Procarbazine	Lomustine (CCNU)	Fotemustine
Mechanism	Alkylating agent (methylating DNA)	Alkylating agent	Alkylating nitrosourea	Alkylating agent (nitrosourea)
Indications	GBM, melanoma	Hodgkin's lymphoma, brain tumors	Brain tumors, melanoma	Brain tumors, melanoma
Administration	Oral	Oral, IV	Oral	IV
Dosing Schedule	Daily x 5 days in 28-day cycle	Varies	Every 3–4 weeks	Weekly or bi-weekly
Market Status	Approved, widely used	Off-label, approved in some markets	Approved	Approved

Temozolomide offers the advantage of oral administration, favorable toxicity profile, and better tolerability, leading to broader adoption.

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Future Outlook and Opportunities

Pipeline and Innovation Opportunities

• New Formulations: Development of sustained-release capsules or injectables to improve adherence.
• Biomarker-Driven Therapy: Use of MGMT methylation status to optimize patient selection.
• Combination Strategies: Further integration with immunotherapy and targeted agents, potentially improving outcomes for resistant tumors.
• Orphan Designation and Expedited Pathways: For pipeline indications, facilitating faster regulatory approval.

Challenges

• Patent Expiries: Increasing generic competition decreasing margins.
• Toxicity Limitations: Managing side effects to optimize therapy adherence.
• Patient Stratification: Necessity to identify responsive subgroups to enhance clinical efficacy.

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Key Takeaways

• Clinical innovations focus on combination therapies, biomarkers, and expanding indications for Temozolomide, which can influence the market trajectory.
• Market growth remains steady, with substantial gains driven by ongoing research, approval of new indications, and global expansion.
• Generic entries are intensifying price competition, but brand loyalty and clinical efficacy maintain demand.
• Emerging opportunities include novel formulations, targeted patient populations, and combination regimens with immunotherapies.
• Risks involve patent expiries, regulatory hurdles, and competition from alternative chemotherapeutics and emerging immuno-oncology agents.

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FAQs

Q1: What are the primary indications for Temozolomide today?
A: The primary indications include newly diagnosed and recurrent glioblastoma multiforme and certain melanomas. Ongoing trials are exploring additional brain tumors and metastatic cancers.

Q2: How does MGMT methylation status impact Temozolomide therapy?
A: MGMT promoter methylation is associated with better response to TMZ, making it a vital factor for personalized treatment planning.

Q3: Are there any significant pipeline drugs similar to Temozolomide?
A: Yes, newer alkylating agents and DNA damage response inhibitors are under development, aiming to improve efficacy and overcome resistance.

Q4: What are the key challenges facing the Temozolomide market?
A: Patent expiries, increasing competition from generics, toxicity management, and resistance development are critical challenges.

Q5: What is the outlook for combination therapies involving Temozolomide?
A: Combining TMZ with immunotherapy agents and tumor-treating fields shows promising results, potentially leading to improved survival rates and broader indications.

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References

1. Grand View Research. (2022). Temozolomide Market Size, Share & Trends Analysis Report.
2. ClinicalTrials.gov. (2023). Current Clinical Trials Involving Temozolomide.
3. FDA and EMA approval summaries for Temozolomide.
4. Recent peer-reviewed articles on TMZ combination therapies and biomarker-based treatment strategies.

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This comprehensive overview equips pharmaceutical professionals, healthcare providers, and market analysts with current insights for strategic decision-making regarding Temozolomide.