CLINICAL TRIALS PROFILE FOR AZITHROMYCIN

505(b)(2) Clinical Trials for Azithromycin

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	NCT00694694 ↗	Azithromycin + Artesunate v Artemether-lumefantrine in Uncomplicated Malaria.	Completed	National Institute for Medical Research, Tanzania	Phase 3	2008-06-01	This trial sets out to determine whether the combination of azithromycin and artesunate (AZ+AS) is as good as the current standard treatment for uncomplicated malaria in Tanzania, artemether-lumefantrine (AL). There are two reasons this is important 1. there are only a limited range of drug combinations which work against malaria in this area of Tanzania 2. azithromycin has antimalarial properties, but is also a broad-spectrum antibiotic, so if the combination is an effective antimalarial it might have a place where there are no diagnostic facilities as syndromic treatment for fever. Artesunate and azithromycin have both been used alone or in combination with other drugs in children in Tanzania for many years, and are considered safe. There is trial evidence for the effectiveness of this combination in adults in Asia, as well as in-vitro (laboratory) evidence that it works against the malaria parasite. The trial randomizes children with non-severe malaria to the new combination AZ+AS or the standard care arm AL. The primary outcome is the parasitological failure rate by day 28- meaning do malaria parasites get cleared, and stay cleared for at least 28 days. Secondary outcomes include safety.
New Combination	NCT00694694 ↗	Azithromycin + Artesunate v Artemether-lumefantrine in Uncomplicated Malaria.	Completed	London School of Hygiene and Tropical Medicine	Phase 3	2008-06-01	This trial sets out to determine whether the combination of azithromycin and artesunate (AZ+AS) is as good as the current standard treatment for uncomplicated malaria in Tanzania, artemether-lumefantrine (AL). There are two reasons this is important 1. there are only a limited range of drug combinations which work against malaria in this area of Tanzania 2. azithromycin has antimalarial properties, but is also a broad-spectrum antibiotic, so if the combination is an effective antimalarial it might have a place where there are no diagnostic facilities as syndromic treatment for fever. Artesunate and azithromycin have both been used alone or in combination with other drugs in children in Tanzania for many years, and are considered safe. There is trial evidence for the effectiveness of this combination in adults in Asia, as well as in-vitro (laboratory) evidence that it works against the malaria parasite. The trial randomizes children with non-severe malaria to the new combination AZ+AS or the standard care arm AL. The primary outcome is the parasitological failure rate by day 28- meaning do malaria parasites get cleared, and stay cleared for at least 28 days. Secondary outcomes include safety.
OTC	NCT01560962 ↗	Efficacy of Over the Counter (OTC) Povidone-Ioldine 5% for Treatment of Acute or Chronic Blepharitis	Terminated	Southern California Institute for Research and Education	N/A	2012-01-01	Objective: To determine the preliminary outcome of external over the counter (OTC) povidone iodine (PI) application in the management of chronic and acute blepharitis vs. currently clinically accepted medical regimen, i.e. eyelid hygiene, antibiotic drops, or antibiotic/steroid ointments. Methodology: One hundred adult patients with chronic and acute blepharitis will be enrolled and randomized into four groups. In group one, 25 patients will be instructed to scrub the lid margin of one eye with 5% PI twice daily for 10 days and the other eye with no intervention. In group two, 25 patients will be instructed to scrub the lid margin of one eye with 5% PI and the other eye will receive warm soaked eyelid wash. In group three, 25 patients will be instructed to scrub the lid margin of one eye with 5% PI and the other eye will receive 1 drop of azithromycin ophthalmic solution twice daily for 10 days. In group four, 25 patients will be instructed to scrub the lid margin of one eye with 5% PI and the other eye will receive tobradex ointment applied to the lid margin. Subjective variables assessed included itchiness, foreign body sensation and eyelid edema (grade 0-4). Objective variables assessed included lid margin redness, meibomian gland plugging and presence/absence of collarets (grade 0-4). Cultures of lid margin at the initiation and at the cessation of treatment were obtained.
New Combination	NCT03431168 ↗	A Novel Regimen to Prevent Malaria and STI in Pregnant Women With HIV	Active, not recruiting	University of Alabama at Birmingham	Phase 2	2018-03-07	More than 3 billion people worldwide are at risk of acquiring malaria and pregnant women living with HIV in Africa are at particular risk. An effective prophylaxis regimen capable of preventing malaria and other common perinatal infections would have great potential to improve adverse birth outcomes. The purpose of this randomized controlled trial is to evaluate a new combination prophylaxis regimen in pregnant women with HIV in Cameroon to determine its efficacy and safety.

All Clinical Trials for Azithromycin

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000617 ↗	Azithromycin and Coronary Events Study (ACES)	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	1998-09-01	To determine whether treatment with azithromycin decreases the rate of coronary heart disease events among patients with stable documented coronary artery disease.
NCT00000641 ↗	A Phase II/III Trial of Rifampin, Ciprofloxacin, Clofazimine, Ethambutol, and Amikacin in the Treatment of Disseminated Mycobacterium Avium Infection in HIV-Infected Individuals.	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	To compare the effectiveness and toxicity of two combination drug treatment programs for the treatment of disseminated Mycobacterium avium infection in HIV seropositive patients. [Per 03/06/92 amendment: to evaluate the efficacy of azithromycin when given in conjunction with either ethambutol or clofazimine as maintenance therapy.] Disseminated M. avium infection is the most common systemic bacterial infection complicating AIDS in the United States. The prognosis of patients with disseminated M. avium is extremely poor, particularly when it follows other opportunistic infections or is associated with anemia. Test tube studies and clinical data indicate that the best treatment program may include clofazimine, ethambutol, a rifamycin derivative, and ciprofloxacin. Test tube and animal studies indicate that amikacin is a bactericidal (bacteria destroying) drug that works better when used with ciprofloxacin. Its role in treatment programs is a key issue because of toxicity and because it must be administered parenterally (by injection or intravenously).
NCT00000811 ↗	A Study to Compare Different Drugs Used to Prevent Serious Bacterial Infections in HIV-Positive Children	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	1969-12-31	This study compares 2 different treatments administered to try to prevent serious bacterial infections (such as pneumonia) in HIV-positive children. A combination of drugs (azithromycin plus atovaquone) will be compared to sulfamethoxazole-trimethoprim (SMX/TMP) alone. This study also evaluates the long-term safety and tolerance of these different drugs. SMX/TMP is a commonly prescribed drug for the prevention of bacterial infections. However, the combination of azithromycin and atovaquone may be safer and more effective than SMX/TMP. This study compares the 2 treatments.
NCT00000811 ↗	A Study to Compare Different Drugs Used to Prevent Serious Bacterial Infections in HIV-Positive Children	Completed	Glaxo Wellcome	Phase 2	1969-12-31	This study compares 2 different treatments administered to try to prevent serious bacterial infections (such as pneumonia) in HIV-positive children. A combination of drugs (azithromycin plus atovaquone) will be compared to sulfamethoxazole-trimethoprim (SMX/TMP) alone. This study also evaluates the long-term safety and tolerance of these different drugs. SMX/TMP is a commonly prescribed drug for the prevention of bacterial infections. However, the combination of azithromycin and atovaquone may be safer and more effective than SMX/TMP. This study compares the 2 treatments.

Clinical Trial Conditions for Azithromycin

Condition Name

Condition Name for Azithromycin
Intervention	Trials
COVID-19	37
HIV Infections	22
Covid19	21
Asthma	17

Condition MeSH

Condition MeSH for Azithromycin
Intervention	Trials
COVID-19	91
Infections	74
Infection	70
Communicable Diseases	58

Clinical Trial Locations for Azithromycin

Trials by Country

Trials by Country for Azithromycin
Location	Trials
United States	832
Japan	71
Brazil	69
Canada	49
China	47

Trials by US State

Trials by US State for Azithromycin
Location	Trials
California	66
Texas	44
North Carolina	39
New York	39
Pennsylvania	38

Clinical Trial Progress for Azithromycin

Clinical Trial Phase

Clinical Trial Phase for Azithromycin
Clinical Trial Phase	Trials
PHASE4	14
PHASE3	5
PHASE2	5

Clinical Trial Status

Clinical Trial Status for Azithromycin
Clinical Trial Phase	Trials
Completed	298
Recruiting	98
Not yet recruiting	54

Clinical Trial Sponsors for Azithromycin

Sponsor Name

Sponsor Name for Azithromycin
Sponsor	Trials
Pfizer	69
National Institute of Allergy and Infectious Diseases (NIAID)	31
University of California, San Francisco	31

Sponsor Type

Sponsor Type for Azithromycin
Sponsor	Trials
Other	1067
Industry	177
NIH	58

Last updated: July 25, 2026

Azithromycin clinical trials update and market projection (2024–2034)

Azithromycin remains a core marketed macrolide with broad respiratory and certain STI indications. The current actionable view is: (1) the clinical-trials pipeline is mostly incremental (new patient populations, optimized regimens, combination strategies, resistance-reduction approaches, and formulation work), (2) market growth is constrained by mature use, generic penetration, and shifting empiric-antibiotic stewardship, and (3) near-term revenue exposure is driven more by geography-specific guideline adherence and stewardship programs than by brand exclusivity. A realistic base-case projection is low-to-mid single-digit value growth through 2034, with volume growth slower and pricing pressure persistent.

What clinical trials are ongoing for azithromycin right now?

Featured snippet answer: Most active trials for azithromycin are evaluating optimized dosing/regimens for common bacterial syndromes, assessing combinations (often with other antibiotics or adjunctive agents), and studying special populations where outcome endpoints differ (pediatrics, older adults, comorbid respiratory disease). The trials that can materially change commercial trajectory are those that support new labels, shorten time-to-therapy, or expand use where resistance or guideline change creates a measurable demand shift.

What trial categories dominate azithromycin studies

• Community-acquired respiratory infections (CAP, acute bronchitis, sinusitis): regimen and duration optimization, relapse and microbiologic eradication endpoints, and comparative designs versus other standard-of-care antibiotics.
• Nontuberculous mycobacteria (NTM), especially MAC: azithromycin-containing multi-drug backbones remain a key area, often tied to outcomes in immunocompromised settings.
• Sexually transmitted infections (STIs): azithromycin trials focus on treatment outcomes, reinfection risk mitigation, and comparison against alternative regimens in guideline-updating contexts.
• Pediatrics and pharmacokinetics/pharmacodynamics (PK/PD): weight-band dosing, safety in younger age groups, and exposure-response modeling.

Trial endpoints that map to payer and guideline adoption

• Clinical cure and microbiologic eradication at prespecified timepoints
• Time to symptom improvement and sustained response
• Resistance selection signals, including macrolide susceptibility outcomes
• Safety signals relevant for real-world prescribing (GI tolerability, QT risk monitoring protocols)

How does azithromycin’s clinical pipeline compare with newer macrolides and alternatives?

Featured snippet answer: Azithromycin’s competitive set increasingly includes newer macrolides and non-macrolide antibiotics, but azithromycin maintains practical advantages: established prescribing patterns, short-course usability, and broad empiric guideline inclusion in many settings. The pipeline tends to be incremental rather than disruptive, so differentiation is mostly via optimization and new use-case evidence rather than a new drug mechanism.

Competitive substitution pressure drivers

• Stewardship and diagnostic stewardship: reduced empiric broad-spectrum use where rapid diagnostics support narrow therapy
• Guideline revisions driven by resistance: macrolide resistance in key respiratory pathogens can reduce empiric attractiveness
• Alternative antibiotic availability: agents with improved spectrum or safety profiles can displace macrolide use in some syndromes

Practical implication for investors and licensors

Pipeline value for azithromycin is most likely to show up as:

• Label expansions in subpopulations where evidence is currently thin
• Regimen changes that can be adopted into local formularies and clinical pathways
• Evidence to maintain appropriateness under stewardship rules

What are the main indications and formulations for azithromycin in the market today?

Featured snippet answer: Azithromycin’s commercial demand is anchored in respiratory infections and select STI treatment. Formulation spread across immediate-release tablets/capsules and oral suspensions supports broad setting use, including outpatient and pediatric prescribing. For NTM, azithromycin use is tied to multi-drug regimens and long-duration therapy, which keeps demand more stable but less “growth-like” than acute, short-course indications.

Indication map (high-level)

• Respiratory infections (common outpatient use)
• STIs (varies by country and guideline, and is sensitive to evolving resistance)
• NTM (MAC) (long-duration combination therapy supports continued baseline demand)

Formulation map

• Oral solid forms for adults
• Oral suspensions for pediatric use
• Regimen formats that enable short-course or long-course scheduling depending on indication

How big is the azithromycin market and what is the revenue mix by region?

Featured snippet answer: The market is mature and heavily generic. Value is driven by volume in respiratory indications and by sustained use in NTM regimens. Regionally, demand tracks population base, guideline patterns, and stewardship enforcement intensity.

Revenue mix structure (typical pattern in mature generics)

• Respiratory infections: high volume, pricing pressure
• NTM: lower volume, higher relative cost per patient-month, often protected by combination evidence even if monotherapies are genericized
• STI: guideline-dependent demand spikes and shifts

When do clinical data typically translate into market share gains for azithromycin?

Featured snippet answer: Evidence most often translates into share gains when it supports either (a) guideline updates in specific geographies or (b) payer/formulary pathway adoption that reduces treatment failures and follow-on prescribing.

Commercial timing mechanics

• Guideline cycle lead times: adoption often lags trial completion by 6 to 24 months
• Formulary decisions: payers respond to endpoints that reduce downstream utilization (relapse, return visits)
• Hospital formularies: procurement is guided by stewardship committees that require local susceptibility context

What are the major market risks for azithromycin through 2034?

Featured snippet answer: The primary risks are resistance-driven guideline displacement, continued price erosion from generic competition, and stewardship limits that reduce empiric macrolide use where resistance or diagnostic clarity supports alternative narrower therapy.

Key risk categories

• Macrolide resistance in respiratory pathogens: reduces perceived appropriateness
• Safety scrutiny: QT prolongation concerns continue to influence prescribing behavior and protocol requirements
• Generic margin compression: sustainability risk for branded remnants and supply-chain participants with less cost advantage

What is the azithromycin market projection from 2024 to 2034?

Base-case (value): low-to-mid single-digit CAGR through 2034, driven by volume stability and incremental expansion in specific use-cases, partially offset by pricing pressure.
Base-case (volume): low single-digit CAGR or flat-to-slight decline in some markets due to stewardship and resistance.

Scenario framework (commercially actionable)

Scenario	Assumptions	Value CAGR (2024–2034)	Volume trend
Bear	Rapid resistance-driven guideline contraction plus sustained price compression	~0% to +2%	Flat to down
Base	Moderate guideline pressure, stable NTM demand, incremental evidence keeps appropriate use	~+2% to +4%	Slight growth or stable
Bull	New evidence expands approved or guideline-supported regimens in underpenetrated populations; pricing stabilizes	~+4% to +6%	Moderate growth

How does generic penetration affect azithromycin economics and pricing?

Featured snippet answer: Azithromycin is a mature, widely genericized antibiotic in most developed markets. Economic outcomes are governed by supply chain cost, quality system strength, and payer contracting mechanics rather than brand exclusivity.

What drives contracted pricing

• Competitor pack size and bioequivalence outcomes in procurement bids
• National tenders and large-account formularies
• Competition intensity and manufacturing capacity expansions in China/India-linked supply networks

What patent landscape exists for azithromycin and why it matters less commercially?

Featured snippet answer: For most mainstream oral azithromycin products, patent barriers are largely historical. Commercial barriers now come from regulatory and quality systems, not new patent exclusivity, except where newer formulations, combination regimens, or specific use claims remain protected in some jurisdictions.

Patent-driven pockets that can matter

• Fixed-dose combinations where relevant data packages or formulation improvements persist
• Special population dosing evidence that can support regulatory updates in select jurisdictions
• Formulation patents (taste masking, stability, or manufacturing process improvements)

How strong is the competitive landscape versus other antibiotics used for respiratory infections?

Featured snippet answer: Competitive displacement is driven by resistance patterns and local guideline choices among beta-lactams, fluoroquinolones, and newer agents. Azithromycin’s place in therapy persists where it aligns with guideline criteria and where pathogen susceptibility supports use.

Competitive comparison lens that matters commercially

• Local susceptibility to macrolides
• Adherence (short-course practicality)
• Safety profile and QT protocol availability
• Total treatment cost including follow-up failure management

What is the R&D opportunity set for azithromycin going forward?

Featured snippet answer: The most plausible commercial uplift is not “new mechanism” R&D. It is evidence generation that helps maintain or expand appropriate use under resistance and stewardship constraints.

High-ROI R&D directions

• Regimen optimization that reduces duration without increasing failure
• Trials in populations where azithromycin retains value (specific respiratory phenotypes, long-course NTM backbones)
• Combination strategies that reduce resistance selection pressure

Key Takeaways

• Azithromycin’s clinical trial activity is largely incremental, centered on optimizing regimens, combination strategies, and evidence in specific populations and syndromes.
• Market trajectory is constrained by mature generic penetration and resistance-driven stewardship pressure, limiting high-growth expectations.
• Through 2034, a realistic base-case is low-to-mid single-digit value growth, with volume roughly stable to mildly positive depending on geography and guideline adherence.
• Commercial upside is most likely to come from evidence that supports guideline and payer pathway adoption, not from brand-like exclusivity.

FAQs

1) Are azithromycin trials focused more on respiratory infections or NTM?
Current trial activity is split, but respiratory and NTM remain the dominant therapy areas because outcome endpoints and standard-of-care comparisons are well defined.

2) Can azithromycin clinical data change empiric prescribing behavior?
Yes when endpoints tie to reduced failure rates and align with stewardship frameworks that incorporate local susceptibility.

3) Will resistance meaningfully reduce azithromycin demand in the next decade?
It can in markets where guideline bodies tighten empiric macrolide use based on resistance signals, but long-duration NTM demand can partially stabilize total use.

4) How do stewardship programs affect azithromycin prescriptions?
They tend to reduce empiric use where rapid diagnostics and resistance context support narrower therapy, while sustaining use in defined scenarios that meet criteria.

5) What type of new evidence is most valuable for azithromycin commercialization?
Evidence supporting label-clarifying or guideline-supporting regimens in under-served populations and regimens that improve clinical cure while minimizing resistance selection.

References

1. No citations were provided in the prompt, and no source materials were supplied for this update.