CLINICAL TRIALS PROFILE FOR EFAVIRENZ

505(b)(2) Clinical Trials for EFAVIRENZ

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	NCT00002234 ↗	Safety and Effectiveness of Giving an Anti-HIV Drug Combination of Adefovir Dipivoxil Plus Didanosine Plus Efavirenz Plus Lamivudine Once Daily to HIV-Infected Patients	Completed	Bristol-Myers Squibb	Phase 2	1969-12-31	The purpose of this study is to see if it is safe and effective to give HIV-infected patients a new combination of anti-HIV drugs taken once daily.
New Combination	NCT00002234 ↗	Safety and Effectiveness of Giving an Anti-HIV Drug Combination of Adefovir Dipivoxil Plus Didanosine Plus Efavirenz Plus Lamivudine Once Daily to HIV-Infected Patients	Completed	Dupont Applied Biosciences	Phase 2	1969-12-31	The purpose of this study is to see if it is safe and effective to give HIV-infected patients a new combination of anti-HIV drugs taken once daily.
New Combination	NCT00002234 ↗	Safety and Effectiveness of Giving an Anti-HIV Drug Combination of Adefovir Dipivoxil Plus Didanosine Plus Efavirenz Plus Lamivudine Once Daily to HIV-Infected Patients	Completed	Glaxo Wellcome	Phase 2	1969-12-31	The purpose of this study is to see if it is safe and effective to give HIV-infected patients a new combination of anti-HIV drugs taken once daily.
New Combination	NCT00002234 ↗	Safety and Effectiveness of Giving an Anti-HIV Drug Combination of Adefovir Dipivoxil Plus Didanosine Plus Efavirenz Plus Lamivudine Once Daily to HIV-Infected Patients	Completed	Gilead Sciences	Phase 2	1969-12-31	The purpose of this study is to see if it is safe and effective to give HIV-infected patients a new combination of anti-HIV drugs taken once daily.

All Clinical Trials for EFAVIRENZ

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000885 ↗	Treatment Success and Failure in HIV-Infected Subjects Receiving Indinavir in Combination With Nucleoside Analogs: A Rollover Study for ACTG 320	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	Group A: To compare the time to confirmed virologic failure (2 consecutive plasma HIV-RNA concentrations of 500 copies/ml or more) between the treatment arms: abacavir (ABC) or placebo in combination with zidovudine (ZDV), lamivudine (3TC), and indinavir (IDV). To evaluate the safety and tolerability of these treatment arms. [AS PER AMENDMENT 06/16/99: To compare the time to confirmed treatment failure, permanent discontinuation of treatment, or death between the treatment arms.] [AS PER AMENDMENT 12/27/01: Groups B, C, and D completed follow-up on March 4, 1999. Therefore, only information pertinent to Group A is applicable.] Group B: To compare the proportion of patients who achieve plasma HIV-1 RNA concentrations below 500 copies/ml, as assessed by the standard Roche Amplicor assay at Week 16, or to compare the absolute changes in plasma HIV-1 RNA concentrations at Week 16 across the treatment arms: ABC or approved nucleoside analogs and nelfinavir (NFV) or placebo in combination with efavirenz (EFV) and adefovir dipivoxil. To compare the safety and tolerability of these treatment arms. Group C: To monitor plasma HIV-1 RNA trajectory over time and determine the time to a confirmed plasma HIV-1 RNA concentration above 2,000 copies/ml on 2 consecutive determinations for patients treated with ZDV or stavudine (d4T) plus 3TC and IDV. Group D: To evaluate plasma HIV-1 RNA responses at Weeks 16 and 48. To evaluate the safety and tolerability of the treatment arms: ABC, EFV, adefovir dipivoxil, and NFV. This study explores new treatment options for ACTG 320 enrollees (and, if needed, a limited number of non-ACTG 320 volunteers) who have been receiving ZDV (or d4T) plus 3TC and IDV and are currently exhibiting a range of virologic responses. By dividing the study into the corresponding, nonsequential cohorts (Groups A, B, C, D), different approaches to evaluating virologic success, i.e., undetectable plasma HIV-1 RNA levels, and virologic failure, i.e., plasma HIV-1 RNA levels of 500 copies/ml or more [AS PER AMENDMENT 12/27/01: 200 copies/ml or more], are explored while maintaining long-term follow-up of ACTG 320 patients. [AS PER AMENDMENT 12/27/01: Groups B, C, and D completed follow-up on March 4, 1999. Therefore, only information pertinent to Group A is applicable. This study will examine the question of whether intensification of therapy can prolong the virologic benefit in individuals whose plasma HIV-1 RNA concentrations have been below the limits of assay detection on ZDV (or d4T) plus 3TC plus IDV.]
NCT00000893 ↗	Safety, Tolerability, and Anti-HIV Activity of DMP 266 (Efavirenz) in Combination With Nelfinavir in HIV-Positive Children	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 1	1997-10-01	Cohort I: The purpose of this study is to see how safe it is to combine 2 anti-HIV medications, efavirenz (EFZ) and nelfinavir (NFV) to treat HIV-positive children and to find an appropriate dose of EFZ to use in combination with NFV. Cohort II: The purpose of this study is to see how safe it is to give EFZ syrup combined with NFV and to measure the levels of EFZ and NFV in the blood. (This purpose reflects a change from the original since there are now 2 different cohorts of patients.) EFZ is an effective anti-HIV medication that easily can be combined with other drugs to treat HIV. This is an early study to determine a safe and effective dose for HIV-positive children. This study also will examine the correct dose of NFV to use in combination with EFZ.
NCT00000893 ↗	Safety, Tolerability, and Anti-HIV Activity of DMP 266 (Efavirenz) in Combination With Nelfinavir in HIV-Positive Children	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1997-10-01	Cohort I: The purpose of this study is to see how safe it is to combine 2 anti-HIV medications, efavirenz (EFZ) and nelfinavir (NFV) to treat HIV-positive children and to find an appropriate dose of EFZ to use in combination with NFV. Cohort II: The purpose of this study is to see how safe it is to give EFZ syrup combined with NFV and to measure the levels of EFZ and NFV in the blood. (This purpose reflects a change from the original since there are now 2 different cohorts of patients.) EFZ is an effective anti-HIV medication that easily can be combined with other drugs to treat HIV. This is an early study to determine a safe and effective dose for HIV-positive children. This study also will examine the correct dose of NFV to use in combination with EFZ.
NCT00000903 ↗	Addition of Efavirenz or Nelfinavir to a Lamivudine/Zidovudine/Indinavir HIV Treatment Regimen	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1969-12-31	To compare time to a virologic failure (first of 2 consecutive plasma HIV RNA levels greater than or equal to 200 copies/ml at or after Week 24) of each 4-drug regimen vs the 3-drug regimen. To determine the safety, tolerance, and virologic benefits of either nelfinavir (NFV) or efavirenz (EFV) with indinavir/lamivudine/zidovudine (IDV/3TC/ZDV) vs IDV/3TC/ZDV alone, in the treatment of patients with advanced HIV disease who have received limited or no prior antiretroviral therapy. Prior ACTG studies have shown that the 3-drug combination regimen (IDV/ZDV/3TC) resulted in improved clinical outcomes and therefore may prolong the effects of therapy. The enhanced effects seen with combination therapies are likely related to a greater suppression of RNA replication and alterations in resistance patterns. Due to the progressive success of combination regimens, it is possible that more potent regimens will further enhance viral suppression and provide more durable treatment responses. In light of the additive suppression of HIV replication determined by pharmacological, immunological, and virological results, nelfinavir (NFV) as an addition to IDV/ZDV/3TC will be evaluated. Based on the potency of nonnucleoside reverse transcriptase inhibitors (NNRTIs) to suppress viral replication and the effectiveness of 3-drug regimens containing NNRTIs, efavirenz (EFV) will also be evaluated as an addition to IDV/ZDV/3TC.
NCT00000912 ↗	A Study on Amprenavir in Combination With Other Anti-HIV Drugs in HIV-Positive Patients	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	The purpose of this study is to compare 4 different combinations of anti-HIV drugs and to determine the number of people whose HIV blood levels decrease to 200 copies/ml or less while on the treatment. This study evaluates the safety of these drug combinations, which include an experimental protease inhibitor (PI), amprenavir. Despite the success that many patients have had with PI treatment regimens, there is still a possibility that patients receiving PIs may continue to have high HIV blood levels. Because of this possibility, alternative drug combinations containing PIs are being studied. It appears that amprenavir, when taken with 3 or 4 other anti-HIV drugs, may be effective in patients with prior PI treatment experience.
NCT00000914 ↗	A Study of the Effectiveness of Different Anti-HIV Treatments in HIV-Positive Individuals Who Have Been on a Protease Inhibitor-Containing Drug Regimen for at Least 16 Weeks	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	The purpose of this study is to compare different treatments for HIV infection to see which works best to lower HIV levels and to raise the number of CD4 cells (cells of the immune system that fight infection), in HIV-positive individuals who have been on a protease inhibitor-containing drug regimen for at least 16 weeks. Researchers have found that combination anti-HIV therapy (multiple drugs given together) can help prevent AIDS-related illnesses and help people with AIDS live longer. In this study, the anti-HIV drug efavirenz (EFV) will be tested with 1 or 2 other protease inhibitors (PIs) to see which combination works best to treat HIV infection. EFV has been shown to limit the amount of HIV virus produced by infected cells.

Clinical Trial Conditions for EFAVIRENZ

Condition Name

Condition Name for EFAVIRENZ
Intervention	Trials
HIV Infections	206
HIV	56
HIV Infection	40
Tuberculosis	33

Condition MeSH

Condition MeSH for EFAVIRENZ
Intervention	Trials
HIV Infections	283
Acquired Immunodeficiency Syndrome	67
Infections	51
Infection	46

Clinical Trial Locations for EFAVIRENZ

Trials by Country

Trials by Country for EFAVIRENZ
Location	Trials
South Africa	87
Spain	82
Canada	80
Thailand	52
United Kingdom	49

Trials by US State

Trials by US State for EFAVIRENZ
Location	Trials
California	98
New York	82
Texas	68
Illinois	67
Florida	64

Clinical Trial Progress for EFAVIRENZ

Clinical Trial Phase

Clinical Trial Phase for EFAVIRENZ
Clinical Trial Phase	Trials
PHASE4	2
PHASE1	5
Phase 4	104

Clinical Trial Status

Clinical Trial Status for EFAVIRENZ
Clinical Trial Phase	Trials
Completed	323
Unknown status	36
Terminated	19

Clinical Trial Sponsors for EFAVIRENZ

Sponsor Name

Sponsor Name for EFAVIRENZ
Sponsor	Trials
National Institute of Allergy and Infectious Diseases (NIAID)	75
Gilead Sciences	45
Bristol-Myers Squibb	28

Sponsor Type

Sponsor Type for EFAVIRENZ
Sponsor	Trials
Other	478
Industry	255
NIH	114

Efavirenz: Clinical Trials Update, Market Analysis, and Projection

Last updated: April 26, 2026

Efavirenz is a first-generation non-nucleoside reverse transcriptase inhibitor (NNRTI) used in HIV-1 treatment and widely available in generic form. Near-term growth is constrained by patent expiry, mature penetration, and regimen shifts toward integrase strand transfer inhibitors (INSTIs), but the drug continues to support baseline demand in established formularies, fixed-dose combinations, and settings where lower-cost regimens remain the dominant procurement driver.

What is the current clinical-trial footprint for efavirenz?

Efavirenz is in late-stage development mainly through lifecycle activity (formulation changes, combination optimization, pediatric evaluation, and pharmacokinetic or adherence-related studies) rather than first-in-class innovation. Trial activity is dominated by investigator-led or sponsor-led work connected to public-health programs, WHO-aligned regimen policies, and generic manufacturers’ bioequivalence and clinical bridging work rather than new molecular entities.

Clinical trial activity pattern (high-level):

• Efavirenz has limited presence in Phase 3 and new pivotal endpoints compared with INSTIs.
• Ongoing studies tend to focus on:
  • Bioequivalence and pharmacokinetics for generic or new fixed-dose combinations
  • Pediatric dosing and safety optimization
  • Adherence, switching, or regimen simplification studies using efavirenz-containing therapy

Implication for R&D and investment:

• Development value is concentrated in product lifecycle, regulatory strategy, and supply competitiveness rather than differentiated efficacy claims.
• Clinical endpoints and label expansion are typically incremental and price-sensitive, especially in markets where efavirenz is entrenched in cost-driven procurement.

Evidence base used for this market view:

• Global regimen guidance places efavirenz in established options but with increased use of INSTIs where feasible.
• WHO consolidated guidance has moved programmatic treatment strategies toward INSTIs for many patient groups, which limits the headroom for new efavirenz claims. (Source: [1], [2])

How do current guideline positions shape efavirenz demand?

WHO consolidated guidelines reflect a regimen shift from NNRTI-based to INSTI-based therapy in many settings, while still recognizing efavirenz-containing regimens as part of treatment landscapes where INSTIs are not yet universally adopted.

Guideline-driven demand impacts:

• Short-term: formularies and national guidelines that already include efavirenz support steady demand.
• Medium-term: growth slows as procurement increasingly prioritizes INSTI regimens in programs that can access them at scale.
• Substitution risk: efavirenz’s primary substitution pathway is within NNRTI strategy categories, not by eliminating HIV treatment demand.

WHO’s guidance is central to procurement behavior in many high-burden countries and informs tender structure, inclusion criteria, and supplier qualification cycles. (Source: [1], [2])

What is efavirenz’s commercial market structure?

Efavirenz has largely transitioned into a mature, genericized market. Competitive dynamics are shaped by:

• Patent status and generic entry leading to commoditization
• Fixed-dose combination supply chains (where efavirenz is used alongside nucleoside backbone agents)
• Government and donor procurement (price, availability, and regulatory compliance drive wins more than differentiation)
• Formulary persistence in stable-care programs

Market composition (what actually moves volume)

1. Generic efavirenz tablets and fixed-dose combinations
2. Procurement-driven purchases by ministries of health, large procurement agencies, and donor-funded programs
3. Switching dynamics: patients may remain on efavirenz until clinical or policy triggers prompt changes

Competitive landscape

• The market is dominated by multi-supplier generic portfolios with tight pricing.
• The key commercial levers are:
  • Supply reliability and manufacturing capacity
  • Regulatory clearance (national registration and quality system performance)
  • Pricing for tender rounds
  • Support for fixed-dose combination contracting and distribution

What do pricing and regimen shifts imply for efavirenz revenue growth?

Efavirenz revenue trajectories are typically defined by two opposing forces:

Downward forces

• Substitution by INSTI-based regimens in guideline-aligned procurement.
• Ongoing generics price competition compressing margins.
• Lifecycle development limited by the mature label and commoditized competitive set.

Offsetting forces

• Existing treatment cohorts and regimen continuity.
• Persistent need for NNRTI options where INSTI affordability, eligibility, or program rollout timing delays adoption.
• Use in fixed-dose combinations where infrastructure and supply contracts already exist.

Net effect: volume stays relatively stable but value grows slowly or declines in nominal terms depending on tender pricing and product mix.

Market projection: how will efavirenz evolve through 2026–2031?

Projection framework (scenario logic)

Efavirenz market direction is best modeled by:

• INSTI adoption rate (faster adoption reduces NNRTI growth)
• Procurement pricing pressure (more generic supply lowers unit value)
• Cohort retention (patients stay on therapy until switching criteria are met)
• Geographic policy heterogeneity (some countries move faster than others)

Base-case projection (directional, decision-grade)

• Volume: modest growth or low-single-digit decline in the absence of major policy setbacks, driven by cohort retention and continued eligibility for NNRTI strategies in constrained settings.
• Value: low to negative growth in mature markets due to generics pricing and tender cycles.
• Profitability: constrained margins because the competitive set is large and efficacy differentiation is not a pricing lever.

This projection is consistent with WHO guidance trends that increasingly emphasize INSTIs for many patient populations, which tends to shift new starts away from NNRTIs over time. (Source: [1], [2])

Where is efavirenz most resilient?

Efavirenz tends to show resilience where:

• NNRTI-based regimens remain entrenched in national treatment protocols.
• INSTI scale-up is slower due to procurement budget, contracting timelines, or supply continuity.
• Fixed-dose combination procurement includes efavirenz-based products for program continuity.

What is the strategic takeaway for R&D and partnering?

For efavirenz, the strategy that creates measurable business value is not new drug innovation but product and access execution:

• Lifecycle clinical work that supports regulatory updates, pediatric dosing claims, or fixed-dose combination changes
• Bioequivalence and quality system differentiation to win tenders
• Supply reliability to protect shelf stability across successive procurement rounds
• Contracting leverage through donor and government program relationships aligned with WHO guidance

Key indicators to monitor for the next tender and policy cycles

1. WHO guideline updates affecting regimen preferred status (new starts vs continuation frameworks) (Source: [1], [2])
2. Adoption pace of INSTI regimens in major high-burden procurement markets
3. Tender price compression and supplier churn in NNRTI product categories
4. Regulatory filing throughput for generic efavirenz and fixed-dose combinations in priority countries
5. Switching program announcements that move cohorts from NNRTI to INSTI classes

Key Takeaways

• Efavirenz is a mature, genericized NNRTI with clinical activity mainly focused on lifecycle and regulatory bridging rather than new pivotal efficacy claims.
• WHO guidance shifts many programs toward INSTIs for many patient groups, limiting headroom for efavirenz market growth on new starts.
• Commercial performance is driven by cohort retention and procurement heterogeneity, supporting stability in volume but constraining value growth through tender price competition.
• R&D and partnering value sits in formulation, fixed-dose combination strategy, pediatric and regulatory work, and supply execution rather than differentiation in clinical efficacy.

FAQs

1) Is efavirenz still recommended by WHO for HIV treatment?
WHO consolidated guidance continues to include NNRTI-based regimens in the treatment landscape, while increasingly emphasizing INSTI-based approaches where feasible and prioritizing patient groups per updated guidance. (Sources: [1], [2])

2) What type of clinical trials dominate for efavirenz today?
Activity is mainly lifecycle oriented, including pharmacokinetic studies, pediatric evaluations, and studies supporting generic or fixed-dose combination approvals rather than new drug-development Phase 3 programs. (Sources: [1], [2])

3) Why is efavirenz market growth limited?
The dominant constraint is regimen switching toward INSTIs in programmatic procurement, combined with generic price compression and mature penetration. (Sources: [1], [2])

4) Where does efavirenz maintain demand most effectively?
Demand remains more resilient in settings where NNRTI regimens are entrenched and INSTI scale-up is slower or procurement constraints delay broader adoption. (Sources: [1], [2])

5) What is the most effective commercialization focus for efavirenz going forward?
Winning relies on supply reliability, regulatory execution, and fixed-dose combination contracting that aligns with national procurement plans and guideline cycles. (Sources: [1], [2])

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References (APA)

[1] World Health Organization. (2021). Consolidated guidelines on HIV prevention, testing, treatment, service delivery and monitoring: Recommendations for a public health approach (2nd ed.). WHO.
[2] World Health Organization. (2023). Consolidated guidelines on HIV prevention, testing, treatment, service delivery and monitoring: Recommendations for a public health approach (updated recommendations). WHO.