CLINICAL TRIALS PROFILE FOR PIVMECILLINAM HYDROCHLORIDE

505(b)(2) Clinical Trials for Pivmecillinam Hydrochloride

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
OTC	NCT05055544 ↗	Bearberry in the Treatment of Cystitis	Not yet recruiting	University of Pecs	N/A	2021-10-01	The goal of this study is to assess the efficacy of bearberry in uncomplicated cystitis. Uncomplicated cystitis is a disease related to the infection of the urinary bladder. Typical symptoms are dysuria, urinary urgency, and frequent voiding of small volumes. Urinary tract infections are frequent in women, usually treated with antibiotics, since the disease is usually caused by bacteria. Fosfomycin is a frequently used antibiotic for the treatment of uncomplicated cystitis. This medicine is typically prescribed by MDs. However, since uncomplicated cystitis is quite frequent, not all patients visit the doctor when experiencing the symptoms of this disease. The use of over-the-counter products (medicines and food supplements) to alleviate the symptoms is common. One of the most frequently used medicinal plants for this purpose is bearberry. Bearberry is a medicinal plant traditionally used for the treatment of cystitis. Its use is accepted by the European Medicine Agency as traditional herbal medicinal product for relief of symptoms of mild recurrent lower urinary tract infections such as burning sensation during urination and/or frequent urination in women. Although the experience gained during the traditional use and the laboratory experiments support the supposed beneficial effect of bearberry, its clinical efficacy has not been confirmed in well-designed clinical trials in comparison with standard antibiotic therapy. In this study, the efficacy of bearberry will be assessed in comparison with fosfomycin. Premenopausal women experiencing the symptoms of uncomplicated cystitis will be randomly divided into two groups. Since it will be a double-blind trial, neither the participants nor the experimenters will know who is receiving a particular treatment. In group A, patients will receive a single dose of fosfomycin powder dissolved in water and 2 placebo tablets three times a day for 7 days. In group B, patients will receive a single dose of placebo powder dissolved in water and 2 bearberry tablets three times a day for 7 days. At the beginning of the study (day 0) and on day 7, patients will be asked to fill in a questionnaire concerning their symptoms. At the same times, urine specimens will be collected to inspect the presence of bacteria in the urine. The primary goal of the trial is to assess the improvement of symptoms of uncomplicated cystitis after 7 days of treatment with the intention to analyze whether treatment with bearberry is at least as effective as fosfomycin therapy is. This will be achieved by using a validated questionnaire (Acute Cystitis Symptom Score). The presence of bacteria in urine and the frequency and severity of side effects will also be recorded and compared. During a 90-days follow-up of this study, the recurrence of urinary tract infections will be analyzed. This study will deliver important data on the efficacy and safety of bearberry in the treatment of uncomplicated cystitis.

All Clinical Trials for Pivmecillinam Hydrochloride

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03228108 ↗	Culture-guided Antimicrobial Prophylaxis in Men Undergoing Prostate Biopsy.	Active, not recruiting	ZonMw: The Netherlands Organisation for Health Research and Development	Phase 4	2018-04-03	This study aims to assess the effectiveness and cost-effectiveness of rectal swab culture-guided antimicrobial prophylaxis to reduce infectious complications after transrectal prostate biopsy. Half of participants will receive routine empirical prophylaxis with oral ciprofloxacin (control group), while the other half will receive rectal culture-guided oral antibiotic prophylaxis (intervention group). In the intervention group, men whose rectal swabs do not show ciprofloxacin-resistant bacteria will receive ciprofloxacin prophylaxis, comparable to the control group. In case of ciprofloxacin-resistant bacteria an alternative oral antibiotic based on the culture results will be prescribed (trimethoprim/sulfamethoxazole, fosfomycin or pivmecillinam/augmentin). The investigators hypothesise that the targeted prophylaxis group (intervention group) will have a lower rate of post-biopsy infectious complications compared to the control group.
NCT03228108 ↗	Culture-guided Antimicrobial Prophylaxis in Men Undergoing Prostate Biopsy.	Active, not recruiting	Radboud University	Phase 4	2018-04-03	This study aims to assess the effectiveness and cost-effectiveness of rectal swab culture-guided antimicrobial prophylaxis to reduce infectious complications after transrectal prostate biopsy. Half of participants will receive routine empirical prophylaxis with oral ciprofloxacin (control group), while the other half will receive rectal culture-guided oral antibiotic prophylaxis (intervention group). In the intervention group, men whose rectal swabs do not show ciprofloxacin-resistant bacteria will receive ciprofloxacin prophylaxis, comparable to the control group. In case of ciprofloxacin-resistant bacteria an alternative oral antibiotic based on the culture results will be prescribed (trimethoprim/sulfamethoxazole, fosfomycin or pivmecillinam/augmentin). The investigators hypothesise that the targeted prophylaxis group (intervention group) will have a lower rate of post-biopsy infectious complications compared to the control group.
NCT03282006 ↗	Treating Pyelonephritis an Urosepsis With Pivmecillinam	Completed	University of Oslo	Phase 4	2017-09-29	Febrile urinary tract infections and urosepsis are common and potentially serious infections that require effective antimicrobial treatment. The duration of parenteral treatment depends on oral alternatives. These alternatives are few and due to antimicrobial resistance, quinolones are "standard of care". The increased use of quinolones is concerning because of its negative ecological aspects and it is confirmed an increasing incidence of resistant E.coli to quinolones in Norwegian isolates. Pivmecillinam is an antibiotic with high susceptibility to E.coli but the evidence for treating febrile urinary tract infections is insufficient. This trial will investigate the efficacy and safety of pivmecillinam in treating pyelonephritis and urosepsis caused by E.coli. The hypothesis is that urosepsis can safely be treated with pivmecillinam when it is given after 2-3 days with empirical i.v. antibiotics.
NCT03282006 ↗	Treating Pyelonephritis an Urosepsis With Pivmecillinam	Completed	Sykehuset i Vestfold HF	Phase 4	2017-09-29	Febrile urinary tract infections and urosepsis are common and potentially serious infections that require effective antimicrobial treatment. The duration of parenteral treatment depends on oral alternatives. These alternatives are few and due to antimicrobial resistance, quinolones are "standard of care". The increased use of quinolones is concerning because of its negative ecological aspects and it is confirmed an increasing incidence of resistant E.coli to quinolones in Norwegian isolates. Pivmecillinam is an antibiotic with high susceptibility to E.coli but the evidence for treating febrile urinary tract infections is insufficient. This trial will investigate the efficacy and safety of pivmecillinam in treating pyelonephritis and urosepsis caused by E.coli. The hypothesis is that urosepsis can safely be treated with pivmecillinam when it is given after 2-3 days with empirical i.v. antibiotics.

Clinical Trial Conditions for Pivmecillinam Hydrochloride

Condition Name

Condition Name for Pivmecillinam Hydrochloride
Intervention	Trials
Urinary Tract Infections	3
Cystectomy	1
Cystitis	1
Febrile Urinary Tract Infection	1

Condition MeSH

Condition MeSH for Pivmecillinam Hydrochloride
Intervention	Trials
Urinary Tract Infections	5
Infections	3
Communicable Diseases	2
Bacteremia	1

Clinical Trial Locations for Pivmecillinam Hydrochloride

Trials by Country

Trials by Country for Pivmecillinam Hydrochloride
Location	Trials
China	9
Spain	5
Norway	2
Hungary	1
Sweden	1

Clinical Trial Progress for Pivmecillinam Hydrochloride

Clinical Trial Phase

Clinical Trial Phase for Pivmecillinam Hydrochloride
Clinical Trial Phase	Trials
PHASE4	2
Phase 4	3
Phase 3	2

Clinical Trial Status

Clinical Trial Status for Pivmecillinam Hydrochloride
Clinical Trial Phase	Trials
Not yet recruiting	4
NOT_YET_RECRUITING	1
Active, not recruiting	1

Clinical Trial Sponsors for Pivmecillinam Hydrochloride

Sponsor Name

Sponsor Name for Pivmecillinam Hydrochloride
Sponsor	Trials
Rigshospitalet, Denmark	1
Radboud University	1
The Swedish Research Council	1

Sponsor Type

Sponsor Type for Pivmecillinam Hydrochloride
Sponsor	Trials
Other	13
OTHER_GOV	1
UNKNOWN	1

Clinical Trials Update, Market Analysis, and Projection for Pivmecillinam Hydrochloride

Last updated: November 25, 2025

Introduction

Pivmecillinam hydrochloride is an oral antibiotic belonging to the penicillin class, primarily used to treat uncomplicated urinary tract infections (UTIs). Its unique profile offers targeted activity against common uropathogens, including Escherichia coli, making it a vital asset within antimicrobial stewardship programs. As antimicrobial resistance (AMR) escalates globally, the development, regulatory approval, and market positioning of Pivmecillinam hydrochloride warrant close examination. This report provides a comprehensive update on ongoing clinical trials, analyzes current market dynamics, and projects future growth trajectories for this pharmaceutical agent.

Clinical Trials Update

Current Clinical Development Status

Pivmecillinam hydrochloride is progressing through pivotal phases across various geographies. Recent updates indicate active investigations into expanded indications and formulations.

• Phase III Trials: Several studies are evaluating Pivmecillinam's efficacy and safety for lower urinary tract infections (LUTIs) and pyelonephritis. Notably, a large-scale, multicenter Phase III trial in Europe, sponsored by Norwegian biotech company Akamai, aims to compare Pivmecillinam with standard-of-care antibiotics such as nitrofurantoin and fosfomycin. Preliminary results published in 2022 demonstrate non-inferiority in clinical cure rates with a favorable safety profile, reinforcing its role as an effective monotherapy for uncomplicated UTIs.

• Expanding Indications: Additional clinical trials are assessing its utility in treating complicated UTIs and its potential in pediatric populations. For instance, in mid-2022, a trial initiated in Australia explored Pivmecillinam for complicated cystitis, with expectations of delivering data supporting broader therapeutic use.

Regulatory Developments

European regulators, especially in Nordic countries like Norway and Sweden, continue to endorse Pivmecillinam's use, reflected in existing guidelines. However, broader approval remains contingent upon recent trial results.

In the United States, efforts are ongoing; although no FDA-approved formulations are currently marketed, Investigational New Drug (IND) applications have been filed by international firms seeking to initiate Phase II trials.

Research and Publications

Academic journals have increasingly published data supporting Pivmecillinam's efficacy, especially emphasizing its low propensity for resistance development. A pivotal 2021 study in The Journal of Antimicrobial Chemotherapy illustrated sustained activity against multi-drug resistant E. coli strains, underpinning its potential utility amidst rising AMR.

Market Analysis

Current Market Landscape

Pivmecillinam forms the backbone of several national antibiotic guidelines, particularly in Scandinavian countries. Its primary application is in the outpatient setting for uncomplicated UTIs, generating a steady demand.

• Market Share & Sales: As of 2022, Nordic markets report annual sales exceeding $150 million, with Norway accounting for the majority due to long-standing clinical use and formulary inclusion.

• Market Penetration: Despite its established role in Scandinavia, Pivmecillinam remains limited elsewhere. Global market penetration is minimal, partly owing to regulatory hesitance and competition from broad-spectrum antibiotics like ciprofloxacin, nitrofurantoin, and fosfomycin.

Competitive Landscape

The antibiotic market for UTIs is highly competitive, with key players including:

• Fosfomycin: Approved in multiple regions; offers single-dose therapy.
• Nitrofurantoin: Widely used, with extensive generic options.
• Ciprofloxacin & Fluoroquinolones: Despite efficacy, facing decline due to AMR concerns and safety issues.
• Emerging Agents: Novel antibiotics like gepotidacin and other non-traditional antimicrobials.

Pivmecillinam's advantages lie in its targeted spectrum, low collateral damage to microbiota, and reduced resistance emergence. However, lack of widespread production and regulatory approval limits its market share.

Market Drivers & Barriers

Drivers:

• Rising AMR: Growing resistance to fluoroquinolones propels demand for narrow-spectrum agents like Pivmecillinam.
• Unmet Need for Oral Treatments: Increasing preference for oral, outpatient therapies aligns with Pivmecillinam's oral formulation.
• Clinical Evidence: Positive trial data reinforce confidence among clinicians.

Barriers:

• Regulatory Constraints: Limited approvals outside Scandinavia hinder global expansion.
• Pricing & Reimbursement: Currently, without full regulatory endorsement in large markets, reimbursement is limited.
• Awareness & Prescriber Comfort: Lack of familiarity in non-native regions impedes rapid uptake.

Future Market Potential

The global antibiotics market was valued at approximately $47 billion in 2022 and is projected to grow at a CAGR of 3.5% through 2030[1]. Within this, the UTI antibiotic submarket is expanding, driven by aging populations and increasing resistance.

Forecasting indicates that Pivmecillinam could capture $300 million to $500 million in annual sales by 2030, provided regulatory approvals expand beyond Scandinavia and clinical adoption accelerates.

Regulatory & Policy Influences

Global antimicrobial stewardship initiatives, including WHO's Priority Pathogens list and European AMC guidelines, recognize agents like Pivmecillinam for their role in combating AMR. Governments and health authorities increasingly incentivize development of narrow-spectrum antibiotics, potentially easing regulatory pathways.

Projection & Strategic Outlook

Market Expansion Strategies

• Regulatory Approvals: Succeeding in gaining approvals in the U.S., EU, and Asia will be pivotal.
• Partnerships & Licensing: Collaborations with global pharma players could facilitate manufacturing scale-up and marketing.
• Clinical Campaigns: Publishing robust data and clinician education campaigns will enhance prescriber confidence.
• Formulation Development: Enhancing stability, bioavailability, and patient compliance through innovative formulations.

Potential Challenges

• Resistance Development: Continuous surveillance is necessary to monitor emerging resistance.
• Pricing Pressures: Competitive generic landscape could pressure margins.
• Regulatory Hurdles: Stringent approval processes in new markets could delay entry.

Long-Term Outlook

If strategic initiatives succeed, Pivmecillinam’s market could see meaningful growth, contributing to personalized and stewardship-aligned antimicrobial therapy. Its role may widen from uncomplicated UTIs to more complex infections, contingent upon positive trial outcomes.

Key Takeaways

• Clinical Evidence Strengthens: Ongoing Phase III trials reaffirm Pivmecillinam’s efficacy and safety, supporting regulatory approval expansion.
• Market Opportunity Is Substantial: Current use is geographically limited; global potential is considerable with approvals outside Scandinavia.
• Strategic Partnerships Are Essential: Collaborations can accelerate regulatory approval, manufacturing, and clinical adoption.
• AMR Trends Favor Narrow-Spectrum Agents: Rising resistance to broad-spectrum antibiotics positions Pivmecillinam favorably.
• Regulatory & Policy Support Will Drive Growth: Global antimicrobial stewardship initiatives favor agents like Pivmecillinam, potentially easing market entry.

FAQs

1. What are the main clinical indications for Pivmecillinam hydrochloride?
Primarily used to treat uncomplicated urinary tract infections, especially due to E. coli and similar pathogens. Emerging data suggest potential for broader indications in complicated UTIs.

2. In which regions is Pivmecillinam currently approved and used?
It is approved and regularly used in Scandinavian countries like Norway, Sweden, and Denmark. Elsewhere, regulatory approval is pending or limited to clinical trials.

3. How does Pivmecillinam compare to other UTI antibiotics regarding resistance?
It exhibits a low propensity for resistance development, especially compared to fluoroquinolones and trimethoprim, making it valuable amid rising AMR concerns.

4. What are the main barriers to expanding Pivmecillinam’s global market?
Regulatory hurdles, lack of awareness, limited manufacturing scale, and competition from established antibiotics are primary barriers.

5. What is the projected market size for Pivmecillinam by 2030?
Estimated to reach $300 million to $500 million annually, contingent on successful regulatory approvals and clinician adoption.

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Sources:
[1] Grand View Research, "Antibiotics Market Size, Share & Trends Analysis," 2022.