CLINICAL TRIALS PROFILE FOR ORLISTAT

505(b)(2) Clinical Trials for Orlistat

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 Dosage	NCT01550926 ↗	A Study to Assess the Pharmacologic Equivalence of Two Orlistat Dosage Forms	Completed	GlaxoSmithKline	Phase 1	2009-02-01	The purpose of this study is to determine whether a new dosage form and dose of orlistat is equivalent to the currently marketed form.

All Clinical Trials for Orlistat

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001723 ↗	Safety and Efficacy of Xenical in Children and Adolescents With Obesity-Related Diseases	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	1998-05-01	Obesity is a condition affecting one-third off the U.S. population and is a major risk actor for the development of Type 2 diabetes, hyperlipidemia (increased levels of fat in the blood), hypertension (high blood pressure), and other disorders of the heart and lungs. Individuals with the onset of obesity during childhood or adolescence are at an increased risk of obesity-related, diseases, both during adolescence and later in adult life. African American girls and women are at an increased risk for obesity, and have substantial rates of obesity-related diseases and causes of death. Further, many African American adult women fail to respond to many of the therapeutic approaches used to treat obesity. At present there are no medical therapies proven effective for the correction of severe obesity in children or adolescents. One medication that may have a favorable risk-benefit ratio in pediatric populations is Orlistat (Xenical, Hoffmann LaRoche). Orlistat works by preventing the action of enzymes in the digestive process, interfering with the absorption of approximately 1/3 of the fat eaten in the diet. Xenical appears to be effective for reducing weight and obesity-associated diseases in obese adults. Researchers propose to determine the safety, tolerability, and efficacy of Xenical in 12-17 year old severely obese African American and Caucasian children and adolescents who have one or more obesity-related disease (hypertension, hyperlipidemia, sleep apnea, hepatic steatosis, insulin resistance, impaired glucose tolerance, or Type 2 diabetes).
NCT00001723 ↗	Safety and Efficacy of Xenical in Children and Adolescents With Obesity-Related Diseases	Completed	Roche Pharma AG	Phase 2	1998-05-01	Obesity is a condition affecting one-third off the U.S. population and is a major risk actor for the development of Type 2 diabetes, hyperlipidemia (increased levels of fat in the blood), hypertension (high blood pressure), and other disorders of the heart and lungs. Individuals with the onset of obesity during childhood or adolescence are at an increased risk of obesity-related, diseases, both during adolescence and later in adult life. African American girls and women are at an increased risk for obesity, and have substantial rates of obesity-related diseases and causes of death. Further, many African American adult women fail to respond to many of the therapeutic approaches used to treat obesity. At present there are no medical therapies proven effective for the correction of severe obesity in children or adolescents. One medication that may have a favorable risk-benefit ratio in pediatric populations is Orlistat (Xenical, Hoffmann LaRoche). Orlistat works by preventing the action of enzymes in the digestive process, interfering with the absorption of approximately 1/3 of the fat eaten in the diet. Xenical appears to be effective for reducing weight and obesity-associated diseases in obese adults. Researchers propose to determine the safety, tolerability, and efficacy of Xenical in 12-17 year old severely obese African American and Caucasian children and adolescents who have one or more obesity-related disease (hypertension, hyperlipidemia, sleep apnea, hepatic steatosis, insulin resistance, impaired glucose tolerance, or Type 2 diabetes).
NCT00001723 ↗	Safety and Efficacy of Xenical in Children and Adolescents With Obesity-Related Diseases	Completed	Jack Yanovski	Phase 2	1998-05-01	Obesity is a condition affecting one-third off the U.S. population and is a major risk actor for the development of Type 2 diabetes, hyperlipidemia (increased levels of fat in the blood), hypertension (high blood pressure), and other disorders of the heart and lungs. Individuals with the onset of obesity during childhood or adolescence are at an increased risk of obesity-related, diseases, both during adolescence and later in adult life. African American girls and women are at an increased risk for obesity, and have substantial rates of obesity-related diseases and causes of death. Further, many African American adult women fail to respond to many of the therapeutic approaches used to treat obesity. At present there are no medical therapies proven effective for the correction of severe obesity in children or adolescents. One medication that may have a favorable risk-benefit ratio in pediatric populations is Orlistat (Xenical, Hoffmann LaRoche). Orlistat works by preventing the action of enzymes in the digestive process, interfering with the absorption of approximately 1/3 of the fat eaten in the diet. Xenical appears to be effective for reducing weight and obesity-associated diseases in obese adults. Researchers propose to determine the safety, tolerability, and efficacy of Xenical in 12-17 year old severely obese African American and Caucasian children and adolescents who have one or more obesity-related disease (hypertension, hyperlipidemia, sleep apnea, hepatic steatosis, insulin resistance, impaired glucose tolerance, or Type 2 diabetes).

Clinical Trial Conditions for Orlistat

Condition Name

Condition Name for Orlistat
Intervention	Trials
Obesity	39
Overweight	8
Overweight or Obesity	4

Condition MeSH

Condition MeSH for Orlistat
Intervention	Trials
Obesity	31
Overweight	17
Weight Loss	13

Clinical Trial Locations for Orlistat

Trials by Country

Trials by Country for Orlistat
Location	Trials
United States	54
China	26
Brazil	6
United Kingdom	6
Sweden	6

Trials by US State

Trials by US State for Orlistat
Location	Trials
Texas	6
North Carolina	5
Ohio	4
New York	4
California	4

Clinical Trial Progress for Orlistat

Clinical Trial Phase

Clinical Trial Phase for Orlistat
Clinical Trial Phase	Trials
PHASE4	4
PHASE3	1
PHASE2	4

Clinical Trial Status

Clinical Trial Status for Orlistat
Clinical Trial Phase	Trials
Completed	38
Recruiting	7
Active, not recruiting	5

Clinical Trial Sponsors for Orlistat

Sponsor Name

Sponsor Name for Orlistat
Sponsor	Trials
GlaxoSmithKline	5
Hoffmann-La Roche	5
Novo Nordisk A/S	4

Sponsor Type

Sponsor Type for Orlistat
Sponsor	Trials
Other	70
Industry	35
NIH	6

Clinical Trials Update, Market Analysis, and Projection for Orlistat

Last updated: October 28, 2025

Introduction

Orlistat, marketed under brand names such as Xenical and Alli, remains a widely prescribed pharmacological intervention for obesity management. As the global obesity epidemic persists, the demand for effective weight-loss therapies sustains interest in existing medications like Orlistat. This report provides a comprehensive update on clinical trials, analyzes current market dynamics, and projects future trends based on recent developments.

Clinical Trials Update for Orlistat

Current Status of Clinical Trials

Orlistat’s clinical development trajectory has shifted from new approvals to comparative efficacy assessments, formulation enhancements, and safety evaluations. According to ClinicalTrials.gov, as of 2023, there are no ongoing late-stage Phase III trials specifically evaluating Orlistat's efficacy or safety, reflecting its established market presence. However, niche studies continue to explore its adjunctive role in metabolic syndrome, type 2 diabetes, and non-alcoholic fatty liver disease (NAFLD).

Emerging Research and Novel Formulations

Recent research emphasizes optimizing Orlistat’s delivery and tolerability:

• Microencapsulated formulations: Aiming to reduce gastrointestinal side effects, which are common deterrents to compliance, by enhancing targeted delivery and stability.
• Combination therapies: Trials investigate synergistic effects when combined with glucomimetics or gut microbiota modulators, seeking to augment weight loss and metabolic benefits.
• Reduced-dose regimens: Studies explore whether lower doses can mitigate gastrointestinal adverse events while maintaining efficacy.

Safety and Efficacy Considerations

Clinical evidence continues to underline Orlistat's safety profile; gastrointestinal disturbances (e.g., steatorrhea, flatulence) remain the most prevalent side effects but are generally manageable. BMI reductions of approximately 5-10% have been documented, with sustained benefits contingent upon lifestyle modifications. Recent trials often include extended follow-ups exceeding two years, indicating a focus on long-term safety and maintenance of weight loss.

Market Analysis

Current Market Overview

The global obesity pharmacotherapy market was valued at approximately USD 2.1 billion in 2022 and is projected to grow at a CAGR of around 7% through 2030, driven by rising obesity prevalence and expanding awareness of pharmacological options. Orlistat maintains a significant share due to its early market entry, affordability, and over-the-counter (OTC) availability in certain markets.

Market Drivers

• Rising obesity rates: Globally, over 650 million adults are obese, fueling demand for effective treatments [1].
• Regulatory support: Increased approvals for obesity medications in Asia and South America expand market access.
• Patient preference for non-systemic treatments: Orlistat's mechanism — inhibiting fat absorption — appeals to patients seeking minimally invasive options.

Competitive Landscape

The market faces competition from newer pharmacotherapies such as:

• GLP-1 receptor agonists (e.g., liraglutide, semaglutide), which demonstrate superior efficacy but are often costlier.
• Combination products: Efforts to combine weight-loss agents with lifestyle supplements or metabolic drugs.

Despite these, Orlistat’s simplicity, low cost, and OTC availability in some regions preserve its relevance, especially in developing markets.

Regulatory and Policy Impact

Regulatory decisions significantly influence market dynamics:

• The FDA's continued approval of OTC Alli ensures broad access.
• Some countries have tightened restrictions due to safety concerns, impacting sales.
• Insurance reimbursement policies favoring newer, more efficacious drugs may challenge Orlistat’s market share.

Market Challenges

• Safety concerns and side effects: gastrointestinal discomfort limits adherence.
• Limited efficacy: Modest weight loss compared to emerging pharmacotherapies.
• Patient perceptions: Preference for newer, "faster" options reduces Orlistat utilization.

Market Projection

Short-term Outlook (2023-2025)

The market will experience steady growth, driven largely by emerging markets and the ongoing obesity crisis. Incremental improvements, such as improved formulations with fewer side effects, are expected to sustain consumer interest. Meanwhile, OTC sales volumes are likely to remain stable, especially in regions with limited healthcare infrastructure.

Medium to Long-term Outlook (2026-2030)

Projected growth may stabilize due to increasing competition from newer, more efficacious agents. However, Orlistat's low cost and established safety profile will preserve its niche, particularly in cost-sensitive settings. The shift towards combination therapies and personalized medicine could open new indications, broadening market applications.

Further innovations, like microencapsulated or sustained-release formulations, are expected to mitigate side effects, expanding patient compliance and long-term use. Regulatory approvals in emerging markets could also act as catalysts for sustained growth, although global market share may decline relative to newer agents, especially in high-income countries.

Key Factors Influencing Future Trends

• Efficacy and safety improvements: Drives patient adherence and physician prescribing.
• Pricing and reimbursement policies: Critical in determining market penetration.
• Patient demographic shifts: Aging populations and rising obesity rates sustain demand.
• Regulatory landscape: Stringent safety evaluations may restrict or expand indications.

Conclusion

Orlistat remains an important weight-loss pharmacotherapy, with ongoing clinical research emphasizing formulation enhancements and combination strategies to improve tolerability and efficacy. While newer drugs threaten its market dominance, Orlistat's low-cost, established safety profile ensure its continued relevance, especially in emerging markets.

Market projections suggest steady growth in the short term, with long-term decline mitigated by incremental improvements and market expansion into underserved regions. However, competitive pressures necessitate continuous innovation and strategic positioning.

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Key Takeaways

• Clinical landscape: Orlistat’s clinical trials focus mainly on safety, tolerability, and combination therapies; no late-stage trials are ongoing as of 2023.
• Market dynamics: It retains significant market share due to low cost and OTC availability, but faces competition from more efficacious, injectable therapies.
• Regulatory influence: Approvals and restrictions in different geographies shape market access and growth opportunities.
• Future outlook: Incremental formulation improvements and expanding markets support steady growth, though long-term market share may decline amid competition.
• Strategic recommendations: Manufacturers should prioritize developing improved formulations, fostering awareness about safety profiles, and targeting emerging markets for sustained revenue.

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FAQs

1. Will Orlistat remain a viable obesity treatment in the next decade?
Yes, particularly in low- and middle-income countries due to its affordability and safety profile. Ongoing innovations and market expansion support its continued relevance.

2. How do new weight-loss drugs compare to Orlistat?
Newer agents, especially GLP-1 receptor agonists, demonstrate greater efficacy with weight losses exceeding 10-15%, but they are often costlier and require injections, unlike Orlistat’s oral, over-the-counter format.

3. Are there successful strategies to improve Orlistat's tolerability?
Yes. Novel formulations such as microencapsulated or sustained-release versions aim to reduce gastrointestinal side effects, potentially improving adherence.

4. What role does regulation play in Orlistat’s future?
Regulatory decisions, including safety assessments and approval of alternative formulations, will influence market access, particularly in regions with stringent policies.

5. What are key areas for research or development to extend Orlistat’s market relevance?
Focus areas include optimizing formulations for fewer side effects, investigating combination therapies for enhanced efficacy, and expanding indications to other metabolic disorders.

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References

[1] World Health Organization. Obesity and overweight. WHO. 2022.