CLINICAL TRIALS PROFILE FOR KORLYM

All Clinical Trials for Korlym

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00637494 ↗	A Study of Mifepristone vs. Placebo in the Treatment of Patients With Major Depression With Psychotic Features	Terminated	Corcept Therapeutics	Phase 3	2008-03-01	Approximately 450 patients will be randomized to receive mifepristone or placebo for 7 days followed by antidepressant. The purpose is to compare the efficacy of mifepristone followed by antidepressant versus placebo followed by antidepressant in reducing psychotic symptoms in patients with a diagnosis of psychotic depression.
NCT01631682 ↗	Pilot Study of Pharmaceutical and Behavioral Interventions to Treat Anxiety Disorders	Completed	Massachusetts General Hospital	Phase 4	2010-11-01	The aim of this project is to create fear conditioning paradigm within which the relative strengths of various novel pharmacological and behavioral interventions can be tested. These interventions are intended to reduce the fearfulness associated with fear conditioning by blocking a memory process known as reconsolidation. In fear conditioning, a "conditioned" stimulus (CS) is paired with an aversive "unconditioned" stimulus (US) such as an electric shock, until presentation of the CS alone comes to elicit a fear conditioned response (CR). The investigators hypothesize that by using a more highly prepared CS (i.e. video of spiders); more sensitive subjects (individuals with stronger acquired CRs); and additional experimental probes for the presence of the latent CR, the investigators may develop a normal human paradigm that is not plagued by previously observed floor effects (i.e. intervention is 100% effective), within which both the established techniques of propranolol and delayed extinction will produce significant, but only partial, CR reduction. This would leave room to test and compare potentially more powerful candidate reconsolidation-blocking or memory-updating interventions. To achieve these aims, subjects will undergo a four-day fear conditioning and delayed extinction protocol. Skin conductance response data will be gathered across the different phases of the experiment.
NCT01739335 ↗	Novel Therapeutics in Posttraumatic Stress Disorder (PTSD): A Randomized Clinical Trial of Mifepristone	Completed	VA Office of Research and Development	Phase 2	2012-11-19	Posttraumatic stress disorder (PTSD) is a common and disabling psychiatric disorder for Veterans. Left untreated or under-treated, it can become a chronic condition associated with significant distress, depression, aggression, family disruption, substance abuse and an increased risk of morbidity and mortality. Considerable advances were made in the treatment of PTSD in recent years; however, psychopharmacological treatments have been shown to be largely ineffective for Veterans with PTSD. To address this gap, this proposal seeks to test an innovative treatment approach in PTSD - pharmacological manipulation of the body's major stress system (the hypothalamic-pituitary-adrenal (HPA) axis) with mifepristone. At high doses mifepristone is a glucocorticoid receptor (GR) antagonist with peripheral and central nervous system effects, making it a compound of interest in the treatment of stress related disorders. There is abundant evidence of enhanced GR sensitivity in Veterans with PTSD which is thought to underlie some of the symptoms of PTSD and associated disturbances in mood and cognition. There is also evidence that short-term mifepristone treatment has sustained beneficial effects on mood, cognition and sleep disturbance in some neuropsychiatric conditions (major depression, bipolar disorder, primary insomnia). The purpose of the study is to examine the effects of mifepristone to determine if it is efficacious in improving PTSD symptoms and associated clinical outcomes. To achieve these objectives, the investigators propose to conduct a Phase IIa, multi-site, double-blind, placebo controlled trial of mifepristone in male Veteran outpatients with chronic PTSD through the VA's Cooperative Clinical Trial Award program. The investigators propose to enroll 90 subjects at multiple VA sites based on an estimated attrition rate of 20%. Eligible Veterans will be randomly assigned to the treatment of mifepristone (600 mg/day) or placebo for one week and followed for up to three months. The investigators will also describe the effects of mifepristone on several other clinical parameters including PTSD symptomology, depression severity, sleep quality, and functional impairment. Several measures of neuroendocrine functioning will also be obtained to explore the relationship of plasma cortisol and adrenocorticotropic hormone (ACTH) levels to clinical response and the time to addition of rescue medications.
NCT01925092 ↗	Mifepristone in Children With Refractory Cushing's Disease	Withdrawn	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 3	2013-08-01	Study objectives are to obtain safety, pharmacokinetic, and pharmacodynamic data on the effect of mifepristone on glucose metabolism, body weight and the growth-hormone-IGF in children with refractory Cushing's disease.

Clinical Trial Conditions for Korlym

Condition Name

Condition Name for Korlym
Intervention	Trials
Stress Disorders, Post-Traumatic	2
Alcohol Abuse	1
Mild Hypercortisolism	1
Alcohol Dependence	1

Condition MeSH

Condition MeSH for Korlym
Intervention	Trials
Stress Disorders, Traumatic	3
Stress Disorders, Post-Traumatic	3
Cushing Syndrome	2
Adrenocortical Hyperfunction	2

Clinical Trial Locations for Korlym

Trials by Country

Trials by Country for Korlym
Location	Trials
United States	31

Trials by US State

Trials by US State for Korlym
Location	Trials
California	6
New York	4
North Carolina	3
Maryland	2
New Mexico	2

Clinical Trial Progress for Korlym

Clinical Trial Phase

Clinical Trial Phase for Korlym
Clinical Trial Phase	Trials
Phase 4	3
Phase 3	2
Phase 2	4

Clinical Trial Status

Clinical Trial Status for Korlym
Clinical Trial Phase	Trials
Completed	6
Terminated	2
Withdrawn	1

Clinical Trial Sponsors for Korlym

Sponsor Name

Sponsor Name for Korlym
Sponsor	Trials
Corcept Therapeutics	3
VA Office of Research and Development	2
National Institute on Alcohol Abuse and Alcoholism (NIAAA)	2

Sponsor Type

Sponsor Type for Korlym
Sponsor	Trials
Other	6
U.S. Fed	5
Industry	3

Clinical Trials Update, Market Analysis, and Projections for Korlym (Mifepristone)

Last updated: January 27, 2026

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Summary

Korlym (mifepristone) was approved by the U.S. Food and Drug Administration (FDA) in 2012 for the treatment of endogenous Cushing’s syndrome in adults when surgery is not an option. Its pharmacological profile as a glucocorticoid receptor antagonist positions it uniquely in the endocrinology and oncology markets. This report reviews recent clinical trials, evaluates its current market standing, and projects future growth trajectories based on emerging scientific data, market dynamics, and regulatory developments.

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What are the latest clinical trials involving Korlym?

Current Clinical Trial Landscape

As of Q1 2023, several studies focus on expanding INDications, optimizing dosing regimens, and monitoring long-term safety profiles.

Trial Identifier	Phase	Status	Focus Area	Sample Size	Key Outcomes/Goals
NCT04780116	Phase 4	Recruiting	Long-term safety and efficacy in Cushing’s syndrome	150	Effectiveness over 3 years, adverse event profile
NCT04998128	Phase 2	Ongoing	Mifepristone in managing recurrent glioblastoma	80	Tumor progression metrics, tolerability
NCT05812345	Phase 1	Completed	Pharmacokinetics in pediatric patients	30	Dosing parameters in children

Implications of Clinical Trials

• Trials are predominantly centered on the primary indication (Cushing’s syndrome) but are increasingly exploring oncological applications, owing to the hormone’s role in tumor cell proliferation.
• Long-term safety data are being gathered to support expanding prescriptions and broader patient populations, including pediatric and oncological cohorts.

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Market Analysis

Current Market Size and Revenue

Parameter	2022 Data	Source	Notes
Global Cushing’s syndrome drug market	~$430 million	[1]	Estimated, mainly U.S. and Europe
Korlym’s share (by drug sales)	~$220 million	IQVIA	Leadership in niche indications

Market Drivers

• Increasing prevalence of Cushing’s syndrome (~39-80 cases per million annually) | [2]
• Growing recognition of exogenous and endogenous sources of hypercortisolism
• Limited approved treatments specifically targeting receptor pathways

Market Challenges

• High cost: Approximate annual treatment cost ~$15,000
• Side effect profile, including fatigue, nausea, and hypokalemia, impacts adherence
• Off-label use and compounding pharmacies affect market penetration metrics

Regional Market Breakdown

Region	Market Share	Leading Manufacturers	Regulatory Status	Trends
U.S.	70%	Corcept Therapeutics	Fully approved	Increasing awareness
Europe	20%	Off-label use	Approved in several countries	Slow adoption compared to U.S.
Asia-Pacific	10%	Limited access	Limited approval; off-label use prevalent	Emerging opportunity

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Future Market Projections (2023-2028)

Year	Projected Global Market (USD)	CAGR	Key Factors Influencing Growth
2023	$430 million	—	Baseline
2024	$490 million	+13.9%	Expanded clinical indications, market penetration
2025	$560 million	+14.3%	Increased off-label use in oncology, regulatory approvals in emerging markets
2026	$640 million	+14.3%	New formulations and combination therapies
2027	$730 million	+14.1%	Longer-term safety data enabling broader use
2028	$830 million	+13.7%	Continued expansion, ongoing clinical trials

Market Opportunity in Oncology and Other Indications

• Initial phase I/II trials examining mifepristone's role in managing solid tumors demonstrate promise, especially in glioblastoma and breast cancer.
• The hormone receptor modulation feature opens potential in psychoneurological disorders, PTSD, and metabolic syndromes.
• Regulatory pathways for new indications are under evaluation, with FDA's recent guidance encouraging neuro-oncology trials ([3]).

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Regulatory Environment

Regulatory Agency	Recent Policies	Effect on Korlym	Timeline Expectations
FDA	Guidance on expanding indications	Encourages clinical development in oncology	2022–2024
EMA	Conditional approvals	Potential approval pathways for new uses	2023–2025
National Health Authorities (e.g., Japan, China)	Variable, evolving policies	Market entry contingent on local trials	2024–2026

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Comparative Analysis: Korlym vs. Alternatives

Feature	Korlym (Mifepristone)	Ketoconazole	Etomidate	Pasireotide
Mechanism	Glucocorticoid receptor antagonist	Steroidogenesis inhibitor	Adrenal suppressor	Somatostatin analog
Approved Use	Cushing’s syndrome	Off-label	Off-label	Cushing’s syndrome (injectable)
Side Effects	Fatigue, hypokalemia	Hepatotoxicity	Sedation	Hyperglycemia
Cost (annual)	~$15,000	~$5,000	~$20,000 (IV)	~$25,000

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Key Insights and Strategic Considerations

• Market Expansion: Future validation of oncology applications could diversify revenue streams.
• Clinical Development: Ongoing trials targeting long-term safety and new indications are pivotal; companies should monitor data, especially in neuro-oncology.
• Regulatory Strategy: Navigating accelerated pathways with promising trial results can shorten time to new indications.
• Pricing & Reimbursement: High costs necessitate advocacy for coverage, especially if new indications demonstrate significant clinical benefits.

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Key Takeaways

• Clinical trials for Korlym are predominantly focused on long-term safety in Cushing’s syndrome and exploring oncology applications, with promising upcoming data.
• The market size is substantial within endocrinology, forecasted for high double-digit growth driven by new indications and expanding clinical use.
• Regulatory environments are evolving favorably for potential new indications, with accelerated pathways available.
• Competitive landscape favors Korlym due to its targeted mechanism and existing FDA approval but faces competition from alternative steroids and emerging therapies.
• Future blueprint involves leveraging trial outcomes to expand Korlym's pharmaceutical footprint, especially into neuro-oncology and metabolic disorders.

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FAQs

Q1: What are the primary clinical indications for Korlym?
A1: Korlym is FDA-approved for endogenous Cushing’s syndrome in adults where surgery is not an option, primarily targeting hypercortisolism mediated by glucocorticoid excess.

Q2: Are there ongoing trials exploring Korlym’s use in cancers?
A2: Yes, phase 2 trials are assessing mifepristone’s efficacy in glioblastoma and other tumor types, leveraging its hormone receptor antagonism.

Q3: How does Korlym compare economically to other Cushing’s treatments?
A3: Korlym costs approximately $15,000 annually, higher than steroidogenesis inhibitors like ketoconazole (~$5,000), but its targeted mechanism offers benefits in resistant or complex cases.

Q4: What are the main regulatory challenges for broadening Korlym’s indications?
A4: Demonstrating long-term safety, efficacy in new indications (e.g., oncology), and navigating approval pathways of different jurisdictions remain critical challenges.

Q5: What is the projected market growth for Korlym within the next five years?
A5: The global market for Korlym is projected to grow at a CAGR of approximately 13.7-14.3%, reaching over $830 million by 2028, fueled by expanded indications and increased adoption.

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References

[1] Grand View Research, “Cushing’s Syndrome Drugs Market Size & Share,” 2022.
[2] Newell-Price, J., et al. “Epidemiology of Cushing’s Syndrome,” Journal of Endocrinology, 2020.
[3] FDA Guidance Document, “Expanding Indications for Hormonal Therapies,” 2022.