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.
OTC	NCT04590274 ↗	Safety and Efficacy of Hydroxychloroquine for the Treatment & Prevention of Coronavirus Disease 2019 (COVID-19) Caused by Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2)	Not yet recruiting	International Brain Research Foundation	Phase 1	2020-11-01	Coronavirus Disease 2019 (COVID-19) (previously called 2019-nCOV acute respiratory disease) is caused by SARS-CoV-2, a positive-sense single-stranded RNA virus of the coronavirus family. The coronaviruses are largely responsible for the common cold, the 2002 SARS outbreak in Guangdong, China, the 2012 MERS outbreak in Saudi Arabia, and the present COVID-19 outbreak that originated in Wuhan, China. Much has been reported by way of systemic injury caused by COVID-19 affecting the cardiovascular, hepatic, nervous systems. These conditions are likely the result of the virus overwhelming the immune system. For these reasons, the investigators wish to conduct this study using existing medications off-label, and over-the-counter supplements to support the immune response, prevent lasting injury, and hasten the recovery from COVID-19.

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.
NCT00000811 ↗	A Study to Compare Different Drugs Used to Prevent Serious Bacterial Infections in HIV-Positive Children	Completed	Pfizer	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	National Institute of Allergy and Infectious Diseases (NIAID)	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
University of California, San Francisco	31
National Institute of Allergy and Infectious Diseases (NIAID)	31

Sponsor Type

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

Azithromycin Clinical Trials Update, Market Analysis, and Projection

Last updated: April 26, 2026

Azithromycin is an established macrolide antibiotic with broad global market penetration and a mature clinical development footprint. Commercial momentum is driven by persistent demand in respiratory and skin/infectious indications, strong generic competition outside a narrow set of branded geographies, and periodic updates to labeling, formulations, and stewardship-driven prescribing patterns. No single “new blockbuster” clinical program is identifiable from the open-ended input provided, but the drug’s near-term market outlook remains anchored by volume stability rather than innovation-led share gains.

What is the current clinical development posture for azithromycin?

Azithromycin’s clinical trial activity is predominantly incremental: formulation refinements, bioequivalence packages, regimen optimization, resistance and epidemiology studies, and guideline-aligned comparative trials. The drug’s key R&D profile is shaped by:

• Entry of multiple generics and the resulting shift of trials toward bridging/bioequivalence or label-adjacent studies rather than novel phase-3 proof of concept.
• Continued investigator-led studies that quantify real-world effectiveness, adherence, and antimicrobial stewardship outcomes.
• Ongoing surveillance and comparative safety work in populations where prescribing is high-volume (pediatrics, community-acquired respiratory infections, and skin/soft tissue settings).

Trial types that dominate

Clinical trial category	Typical endpoints	Market relevance
Comparative effectiveness trials	Clinical cure, microbiologic eradication, symptom scores	Supports guideline-concordant use and payer coverage
Safety and tolerability studies	QT assessment, GI tolerability, adverse event rates	Influences formulary placement and risk-management language
PK/PD, regimen optimization	Concentration-time profiles, adherence-related endpoints	Maintains prescriber confidence in dosing regimens
Formulation and bioequivalence	Cmax, Tmax, AUC, equivalence bounds	Enables generic launches and reduces branded share drag in some segments

Which clinical trial updates materially affect commercial positioning?

Across mature antibiotics, the commercial “signal” comes less from new mechanisms and more from changes that tighten real-world prescribing and reimbursement logic:

• Expanded or clarified label language for specific respiratory and skin indications based on surveillance and comparative evidence.
• Safety messaging tied to cardiac risk, including QT prolongation considerations and drug interaction guidance.
• Regimen simplification studies that reduce treatment duration or support fixed dosing strategies, improving adherence and reducing discontinuation-driven failures.

These factors typically influence channel behavior (physicians, pharmacists, and payers) without changing the underlying market structure.

How big is the azithromycin market and what is the demand engine?

Azithromycin is a high-volume antibiotic in outpatient and community settings. Demand is supported by:

• Persistent burden of community-acquired respiratory infections and common skin/soft tissue infections.
• Routine use patterns in primary care and urgent care, where prescribers favor familiar oral regimens.
• Ongoing generic availability that sustains broad access and keeps utilization high.

Market structure: brand vs generic

Azithromycin is widely generic. Branded revenue is concentrated in geographies or lifecycle pockets where exclusivity still applies to specific products (formulations, branded packs, or local regulatory statuses), while generic competition dominates pricing globally.

Demand drivers vs headwinds

Demand drivers	Commercial headwinds
High baseline prescription volume	Antimicrobial stewardship restrictions and diagnostic testing adoption
Broad labeled indications in respiratory and skin	Competition from newer classes (respiratory fluoroquinolone alternatives, newer macrolides in specific settings)
Oral convenience and familiar dosing	Rising resistance prevalence in certain pathogens shifts empiric choices
Pediatric and outpatient convenience	Safety constraints in at-risk cardiac populations

What does the pricing and margin outlook look like?

Azithromycin pricing compresses with generic penetration. The margin story is defined by:

• Competitive procurement pricing in large markets.
• Tender and hospital formulary leverage lowering net pricing.
• Brand premium only where limited branded supply or specialty formulation differentiates procurement.

For investors and R&D planners, the practical implication is that incremental clinical activity typically does not translate into durable pricing power. Market share gains, where they occur, come from supply reliability, distribution contracts, and specific formulation advantages rather than from new mechanism-driven superiority.

What are the most relevant competitive dynamics for azithromycin?

Substitution and category competition

Azithromycin competes within the macrolide class and across antibiotics used for similar empiric indications:

• Other macrolides (where relevant to local guidelines or tolerability profiles)
• Cephalosporins and penicillin combinations (respiratory and skin pathways)
• Fluoroquinolones for specific adult populations and guideline-selected cases
• Doxycycline and other alternatives in stewardship-led decision trees where appropriate

Resistance-driven prescribing shifts

Resistance patterns to common azithromycin-relevant pathogens (notably respiratory and skin-associated bacteria in certain regions) influence empiric use. The effect on commercial outlook is indirect but real: stewardship and diagnostic strategies can reduce unnecessary prescribing, and local resistance data can move first-line choices toward other classes.

Market projection: what changes from 2026 onward?

A mature antibiotic’s projection depends on two axes: utilization and price. For azithromycin, utilization remains structurally supported but is bounded by stewardship. Price continues a downward slope in highly generic markets, with relative stability in places where procurement contracts lock pricing.

Projection framework (directional)

Variable	Near-term direction	Rationale
Global utilization	Flat to modestly positive	Persistent infection burden and high outpatient use
Net pricing	Flat to down	Continued generics and procurement pressure
Revenue growth	Low single-digit at best	Offset utilization stability against price erosion
Brand premiums	Limited	Generic baseline limits durable branded advantage

What does this mean for R&D strategy and investment screening?

For azithromycin specifically, the highest-value R&D is typically:

• Formulation and patient-experience optimization (dose adherence, taste/dispersion where pediatric use matters).
• Safety refinement and label optimization tied to interaction/QT-risk mitigation.
• Rapid bridging studies that enable lifecycle extension in specific markets where branded or semi-branded products remain advantaged.

For investment screening, the “signal” tends to be supply-side and regulatory execution rather than major clinical novelty. In practice, the best opportunities in mature antibiotics usually sit in:

• Securing distribution and tender placement.
• Defending a formulation niche where switching costs or procurement preferences exist.
• Using regulatory submissions strategically to protect share in a specific geography.

Key Takeaways

• Azithromycin clinical activity is mature and incremental, dominated by regimen, formulation, and evidence-aligned studies rather than mechanism-changing innovation.
• Market performance remains demand-anchored by common outpatient indications, with stewardship and resistance patterns acting as constraints on utilization growth.
• Revenue growth is structurally capped by generic-driven pricing compression; future gains, if any, come from supply and formulation niches rather than durable pricing power.
• Near-term outlook is best modeled as stable utilization with gradual price erosion, translating into low single-digit revenue growth in broad projections, depending on regional procurement dynamics.

FAQs

1. Is azithromycin still under active clinical investigation?
   Yes, but activity is predominantly incremental, including comparative, formulation, safety, PK/PD, and stewardship-aligned evidence studies.

2. What drives azithromycin demand today?
   Persistent outpatient infection burden in respiratory and skin/soft tissue pathways and routine prescribing where diagnostic and guideline practices still support empiric use.

3. How does generic competition affect azithromycin’s market outlook?
   It compresses net pricing and limits branded revenue durability, making growth dependent on utilization stability and procurement dynamics.

4. Do resistance trends materially change azithromycin’s commercial trajectory?
   They can reduce empiric selection in specific regions and pathogens, and they strengthen stewardship controls that can lower unnecessary prescribing.

5. What type of new product would be most commercially relevant for azithromycin?
   Patient-adherence and formulation improvements, and label refinements that preserve guideline-concordant positioning without triggering major payer or safety switches.

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References

[1] FDA. Drug Approval Packages: Azithromycin. U.S. Food and Drug Administration. https://www.accessdata.fda.gov/scripts/cder/daf/
[2] European Medicines Agency (EMA). Azithromycin: EPAR and related information. European Medicines Agency. https://www.ema.europa.eu/
[3] World Health Organization (WHO). WHO Model List of Essential Medicines: azithromycin and antibiotic use guidance. World Health Organization. https://www.who.int/
[4] Antimicrobial Resistance Collaborators. Resistance patterns and antibiotic use context (global synthesis). The Lancet (series on AMR). https://www.thelancet.com/