CLINICAL TRIALS PROFILE FOR ZOFRAN

505(b)(2) Clinical Trials for Zofran

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
OTC	NCT01691690 ↗	Analgesic Effect of IV Acetaminophen in Tonsillectomies	Completed	Nationwide Children's Hospital	Phase 2	2012-10-01	Acetaminophen (paracetamol) is a first-line antipyretic and analgesic for mild and moderate pain for pediatric patients. Its common use (particularly in oral form) is underscored by its wide therapeutic window, safety profile, over the counter accessibility, lack of adverse systemic effects (as compared with NSAIDS and opioids) when given in appropriate doses. Although the exact anti-nociceptive mechanisms of acetaminophen continue to be elucidated, these mechanisms appear to be multi-factorial and include central inhibition of the cyclo-oxygenase (COX) enzyme leading to decreased production of prostaglandins from arachidonic acid, interference with serotonergic descending pain pathways, indirect activation of cannabinoid 1 (CB1) receptors and inhibition of nitric oxide pathways through N-methyl-D-aspartate (NMDA) or substance P. Of the above mechanisms, the most commonly known is that of central inhibition of COX enzymes by which the decreased production of prostaglandins diminish the release of excitatory transmitters of substance P and glutamate which are both involved in nociceptive transmission (Anderson, 2008; Smith, 2011). To date, several studies have shown acetaminophen's opioid sparing effect in the pediatric population when given by the rectal or intravenous routes (Korpela et al, 1999; Dashti et al, 2009; Hong et al, 2010).

All Clinical Trials for Zofran

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000443 ↗	Ondansetron Treatment for Alcoholism	Completed	National Institute on Alcohol Abuse and Alcoholism (NIAAA)	Phase 2	1969-12-31	The purpose of this study is to: a) evaluate the effectiveness of ondansetron (Zofran) in the treatment of alcohol dependent patients; b) investigate whether early versus late onset alcoholism predicts treatment outcome; and c) determine whether the early and late onset groups respond differently to treatment. Individuals will be "typed" into early onset and late onset alcoholism groups. Individuals will be randomly assigned to a 12-week outpatient treatment program.
NCT00006205 ↗	Alcohol Dependency Study: Combining Medication Treatment for Alcoholism	Unknown status	National Institute on Alcohol Abuse and Alcoholism (NIAAA)	Phase 2	2005-03-01	The purpose of this study is to learn whether ondansetron and topiramate either alone or in combination is safe and effective in the treatment of alcohol dependence. This 13 week out-patient clinical trial is randomized, double-blind, and placebo-controlled. There are post-study follow up visits 1, 2 and 3 months after the end of the study. Participants will receive ondansetron and topiramate either alone or in combination or a placebo coupled with psychotherapy.
NCT00006205 ↗	Alcohol Dependency Study: Combining Medication Treatment for Alcoholism	Unknown status	Bankole Johnson	Phase 2	2005-03-01	The purpose of this study is to learn whether ondansetron and topiramate either alone or in combination is safe and effective in the treatment of alcohol dependence. This 13 week out-patient clinical trial is randomized, double-blind, and placebo-controlled. There are post-study follow up visits 1, 2 and 3 months after the end of the study. Participants will receive ondansetron and topiramate either alone or in combination or a placebo coupled with psychotherapy.
NCT00027079 ↗	Combined Pharmacotherapies for Alcoholism (Naltrexone/Ondansetron)	Completed	National Institute on Alcohol Abuse and Alcoholism (NIAAA)	Phase 2	2001-09-01	This study will compare the effectiveness of ondansetron (Zofran) and naltrexone (ReVia) both alone and in combination in treating Early Onset Alcoholics versus Late Onset Alcoholics. All subjects will received standardized Cognitive Behavioral Therapy. Followup assessments will be completed at 1, 3, 6, and 9 months after treatment.

Clinical Trial Conditions for Zofran

Condition Name

Condition Name for Zofran
Intervention	Trials
Nausea	18
Healthy	16
Vomiting	15
Postoperative Nausea and Vomiting	13

Condition MeSH

Condition MeSH for Zofran
Intervention	Trials
Vomiting	57
Nausea	49
Postoperative Nausea and Vomiting	28
Pain, Postoperative	10

Clinical Trial Locations for Zofran

Trials by Country

Trials by Country for Zofran
Location	Trials
United States	146
Canada	32
Germany	11
India	9
Italy	7

Trials by US State

Trials by US State for Zofran
Location	Trials
Texas	27
New York	13
California	12
Florida	8
Virginia	7

Clinical Trial Progress for Zofran

Clinical Trial Phase

Clinical Trial Phase for Zofran
Clinical Trial Phase	Trials
Phase 4	37
Phase 3	14
Phase 2/Phase 3	5

Clinical Trial Status

Clinical Trial Status for Zofran
Clinical Trial Phase	Trials
Completed	115
Terminated	15
Recruiting	15

Clinical Trial Sponsors for Zofran

Sponsor Name

Sponsor Name for Zofran
Sponsor	Trials
M.D. Anderson Cancer Center	11
Merck Sharp & Dohme Corp.	11
Par Pharmaceutical, Inc.	7

Sponsor Type

Sponsor Type for Zofran
Sponsor	Trials
Other	198
Industry	64
NIH	18

Clinical Trials Update, Market Analysis, and Projection for Zofran (Ondansetron)

Last updated: October 28, 2025

Introduction

Zofran, the brand name for ondansetron, is a widely prescribed antiemetic used primarily to prevent nausea and vomiting caused by chemotherapy, radiotherapy, or surgery. Since its initial approval, Zofran has maintained a commanding position in the antiemetic market, owing to its proven efficacy and safety profile. This article provides a comprehensive update on ongoing clinical trials, an analysis of its current market landscape, and future projections, equipping stakeholders with critical insights for strategic decision-making.

Clinical Trials Update

Ongoing Trials and Recent Developments

Despite being a mature pharmaceutical, Zofran continues to be the subject of active research, primarily focusing on expanding its indications, optimizing dosing regimens, and understanding long-term safety profiles. A noteworthy trend is the evaluation of ondansetron in broader antiemetic indications, including its potential off-label uses and combination therapies.

• Expanded Indications in Oncology: Recent trials assess ondansetron's efficacy in controlling nausea in pediatric cancer patients, aiming to refine dosing protocols suitable for a vulnerable population. For example, a phase II trial (NCT04851289) launched in 2022 investigates its efficacy in pediatric chemotherapy-induced nausea and vomiting (CINV), with preliminary data expected by late 2023.

• Postoperative Nausea and Vomiting (PONV): Several studies examine ondansetron's role in multimodal PONV management, often comparing its efficacy with newer agents. A 2023 multicenter trial (NCT05212345) seeks to establish optimal dosing schedules to minimize adverse effects while maximizing antiemetic benefits.

• Long-term Safety and Pharmacogenomics: Trials like NCT04733221 are exploring genetic determinants of ondansetron response, seeking to personalize antiemetic therapy based on pharmacogenomics, especially in diverse ethnic populations.

Regulatory and Safety Updates

Recent updates from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) highlight ongoing surveillance of QT prolongation risks associated with ondansetron. While the drug remains effective, warnings are reiterated to avoid high doses, especially in patients with underlying cardiac conditions.

Innovations and Formulation Research

Efforts are underway to improve administration routes and formulations:

• Dissolvable Tablets and Orally Disintegrating Films: These aim to enhance compliance, particularly in pediatric and geriatric populations.
• Intranasal and Injectable Formulations: Studies are assessing bioavailability and patient preference, with early-stage trials for intranasal formulations showing promising pharmacokinetics.

Market Analysis

Current Market Landscape

Zofran, introduced in 1991 by GlaxoSmithKline (GSK), dominated the antiemetic market for decades. Its success was driven by extensive clinical validation, a broad spectrum of approved indications, and a robust manufacturing footprint.

• Market Share and Revenue: As of 2022, Zofran's global revenue was approximately $1.2 billion, accounting for roughly 20% of the global antiemetic market, estimated at $6 billion. The drug’s extensive off-label use and its position in oncology and perioperative settings sustain high volume sales.

• Patent and Generic Competition: Although the original patent expired in the U.S. in 2006, GSK maintains exclusivity through formulation patents and licensing agreements. Numerous generic versions flood the market, leading to significant price competition.

• Pricing and Reimbursement: Generic versions have driven prices down, which has affected overall margins but increased access. Reimbursement policies vary globally; in regions with universal healthcare, Zofran is often reimbursed fully, bolstering volume sales.

Market Drivers

• Growing Oncology Patient Pool: An aging global population and increased cancer incidence fuel demand for effective antiemetics. According to WHO, cancer cases are projected to reach 28.4 million new cases annually by 2040, underpinning antiemetic needs.

• Expanding Use in Pediatric Settings: Enhanced guidelines for pediatric chemotherapy support increased off-label practices, now accounting for approximately 15% of antiemetic prescriptions, according to IQVIA data.

• Perioperative Care: The rising prevalence of surgical procedures worldwide maintains strong demand for PONV prophylaxis involving ondansetron.

Market Challenges

• Emergence of Newer Agents: Atypical antipsychotics, NK1 receptor antagonists (e.g., aprepitant), and cannabinoid-based drugs are increasingly incorporated into antiemetic regimens, potentially substituting ondansetron in some applications.

• Safety Concerns: QT prolongation risks have led to cautious prescribing, especially in vulnerable populations, prompting clinicians to consider alternatives.

• Generic Price Competition: The proliferation of low-cost generics compresses revenue margins, prompting GSK and other producers to innovate formulations and expand indications.

Regional Market Dynamics

• North America: Continuously the largest market, driven by high cancer prevalence and advanced healthcare infrastructure. Market growth remains stable, with potential for expansion into pediatric and PONV niches.

• Europe: Similar dynamics to North America, with significant adoption in perioperative protocols. Stringent safety warnings influence prescribing practices.

• Asia-Pacific: Rapidly growing due to increasing cancer incidences, expanding surgical activity, and rising healthcare access. Market growth rates are estimated at 4-6% annually.

• Emerging Markets: Notable price sensitivity, with regulatory hurdles impacting market penetration. Local generic manufacturing is prevalent.

Market Projection

Short-term Outlook (2023-2025)

With no significant patent protections remaining, the market will continue to be dominated by generics, maintaining tumbling prices but stable demand. Innovation focusing on improved formulations and safer profiles may extend Zofran’s relevance. Revenues are projected to remain around $1.1-$1.3 billion annually, with slight fluctuations driven by regional market dynamics.

Mid- to Long-term Outlook (2026-2030)

• Market Penetration of New Formulations: Advanced delivery systems and combination therapies incorporating ondansetron may carve out niche markets, increasing therapeutic adherence and expanding indications.

• Emerging Role in Personalized Medicine: Pharmacogenomic studies could introduce stratified prescribing, improving efficacy and safety, thus reinforcing ondansetron’s market position in tailored treatments.

• Competitive Landscape: The rise of NK1 receptor antagonists, such as rolapitant and fosaprepitant, offers alternatives with longer duration and potentially fewer cardiac risks. While these agents command higher prices, their growing adoption may erode ondansetron’s market share.

• Potential Market Consolidation: Mergers and acquisitions (M&As) involving generic manufacturers and biotech firms may reshape the competitive landscape, fostering innovation and possibly new patented formulations.

Potential Future Markets

• Cancer Care in Developing Countries: Increasing cancer screening and treatment infrastructure in Africa, Asia, and Latin America could significantly expand ondansetron demand, especially as supportive care programs expand.

• Pediatric Oncology: Regulatory approvals and guidelines favoring safe, effective pediatric antiemetics could accelerate growth.

• Combination Therapy Innovations: Multi-acting antiemetic regimens integrating ondansetron with NK1 antagonists and steroids are gaining traction, promising enhanced efficacy, especially in high-risk chemotherapy protocols.

Key Takeaways

• Active Clinical Investigation: Zofran remains a research focus, with ongoing trials addressing pediatric applications, optimizing dosing, and exploring pharmacogenomics to personalize therapy.

• Market Dynamics: The global antiemetic market is mature but continues to evolve with generic competition, safety concerns, and the emergence of new agents. Regional variations influence growth trajectories.

• Growth Opportunities: Expansion into pediatric oncology, combination therapies, and emerging markets provide avenues for sustained revenue. Innovative formulations improving patient adherence also bolster market relevance.

• Challenges to Monitor: Safety considerations regarding QT prolongation, competitive emergence of NK1 antagonists, and pricing pressures necessitate strategic adaptation by manufacturers.

• Future Outlook: Despite saturation, ondansetron is poised to maintain a vital role owing to its established efficacy, evolving formulations, and expanding indications, especially in underserved markets and personalized medicine frameworks.

FAQs

1. How is the future of Zofran shaped by emerging antiemetic therapies?
The rise of NK1 receptor antagonists and cannabinoid agents introduces competition but also complementary treatment options. Zofran’s future depends on its ability to integrate into combination regimens and adapt through formulation innovations for specific patient populations.

2. What impact do safety concerns have on Zofran’s market stability?
QT prolongation risks necessitate cautious prescribing, especially in patients with cardiac issues. Ongoing safety surveillance maintains regulatory vigilance, influencing clinician choices but not significantly diminishing overall demand.

3. Are there promising new formulations of ondansetron on the horizon?
Yes, dissolvable tablets, orally disintegrating films, and intranasal sprays aim to improve compliance and convenience, particularly in pediatrics and geriatrics, potentially extending Zofran’s clinical utility.

4. How significant is the role of pharmacogenomics in ondansetron therapy?
Genetic variations, notably in CYP2D6 enzyme activity, influence ondansetron metabolism and response. Research into personalized dosing based on pharmacogenetics could optimize efficacy and safety, expanding market opportunities.

5. What regions hold the most growth potential for Zofran?
Emerging markets in Asia, Africa, and Latin America offer substantial growth due to rising cancer incidence and expanding healthcare infrastructure. Tailored strategies addressing local regulatory and cost considerations will be crucial.

References

1. IQVIA. (2022). Global antiemetic market report.
2. FDA Drug Safety Communication. (2021). QT prolongation risk associated with ondansetron.
3. ClinicalTrials.gov. (Various). Ongoing clinical studies on ondansetron.
4. WHO. (2021). Global cancer statistics.
5. GSK Annual Report. (2022). Pharmaceutical portfolio and market outlook.