CLINICAL TRIALS PROFILE FOR PENICILLIN V

505(b)(2) Clinical Trials for Penicillin V

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 Indication	NCT05069974 ↗	Alternative Antibiotics for Syphilis	Recruiting	Fundación FLS de Lucha Contra el Sida, las Enfermedades Infecciosas y la Promoción de la Salud y la Ciencia	Phase 3	2021-10-01	The Trep-AB clinical trial will test the efficacy of an investigational neuropenetrative drug, Linezolid (LZD), compared to standard treatment, Benzathine penicillin G (BPG), for early syphilis in humans. The overarching idea of the work proposed herein is to investigate the use of LZD to treat syphilis, conducting a randomized controlled clinical trial to evaluate this new indication of a known antibacterial agent. It is estimated to include 360 participants.

All Clinical Trials for Penicillin V

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000585 ↗	Penicillin Prophylaxis in Sickle Cell Disease (PROPS)	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	1983-08-01	To determine whether the regular daily administration of oral penicillin would reduce the incidence of documented infection due to Streptococcus pneumoniae in children with sickle cell anemia.
NCT00000648 ↗	A Pilot Study Evaluating Penicillin G and Ceftriaxone as Therapies for Presumed Neurosyphilis in HIV Seropositive Individuals	Completed	Hoffmann-La Roche	N/A	1969-12-31	To provide information on the response of HIV infected, neurosyphilis patients to the currently recommended treatment for neurosyphilis; to determine whether possible co-infection with both HIV and syphilis makes more difficult the diagnosis of syphilis; to explore the usefulness of an alternative treatment which, if effective, would permit outpatient treatment for neurosyphilis that until now required prolonged hospitalization. Studies suggest that syphilis treatment failures may be more common in HIV infected patients than in patients without HIV infection and that treatment failures occur due to and/or are displayed as central nervous system (CNS) involvement. Very little is known about the best treatment course for neurosyphilis in patients who are also infected with HIV.
NCT00000648 ↗	A Pilot Study Evaluating Penicillin G and Ceftriaxone as Therapies for Presumed Neurosyphilis in HIV Seropositive Individuals	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	To provide information on the response of HIV infected, neurosyphilis patients to the currently recommended treatment for neurosyphilis; to determine whether possible co-infection with both HIV and syphilis makes more difficult the diagnosis of syphilis; to explore the usefulness of an alternative treatment which, if effective, would permit outpatient treatment for neurosyphilis that until now required prolonged hospitalization. Studies suggest that syphilis treatment failures may be more common in HIV infected patients than in patients without HIV infection and that treatment failures occur due to and/or are displayed as central nervous system (CNS) involvement. Very little is known about the best treatment course for neurosyphilis in patients who are also infected with HIV.
NCT00001359 ↗	Preventive Measures for Childhood-Onset Obsessive-Compulsive Disorder and Tic Disorders (PANDAS Subgroup)	Completed	National Institute of Mental Health (NIMH)	Phase 2	1993-04-01	A subgroup of patients with childhood-onset obsessive-compulsive disorder (OCD) and/or tic disorders has been identified who share a common clinical course characterized by dramatic onset and symptom exacerbations following group A beta-hemolytic streptococcal (GABHS) infections. This subgroup is designated by the acronym PANDAS (Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal infections). There are five clinical characteristics that define the PANDAS subgroup: presence of OCD and/or tic disorder; prepubertal symptom onset; sudden onset or abrupt exacerbations (relapsing-remitting course); association with neurological abnormalities (presence of adventitious movements or motoric hyperactivity during exacerbations); and temporal association between symptom exacerbations and GABHS infections. In this subgroup, periodic exacerbations appear to be triggered by GABHS infections in a manner similar to that of Sydenham's chorea, the neurological variant of rheumatic fever. Rheumatic fever is a disorder with a presumed post-streptococcal autoimmune etiology. The streptococcal pathogenesis of rheumatic fever is supported by studies that have demonstrated the effectiveness of penicillin prophylaxis in preventing recurrences of this illness. A trial of penicillin prophylaxis in the PANDAS subgroup demonstrated that penicillin was not superior to placebo as prophylaxis against GABHS infections in these children, but this outcome was felt to be secondary to non-compliance with treatment, and there was no decrease in the number of neuropsychiatric symptom exacerbations in this group. In a study comparing azithromycin and penicillin, both drugs were completely effective in preventing streptococcal infections - there were no documented titer elevations during the year-long study period for children taking either penicillin or azithromycin. Comparable reductions in the severity of tics and obsessive-compulsive symptoms were also observed. Thus, penicillin was not performing as an "active placebo" as originally postulated, but rather provided effective prophylaxis against Group A beta-hemolytic streptococcal. Both azithromycin and penicillin appear to be effective in eliminating GABHS infections, and reducing neuropsychiatric symptom severity; thus, between-group differences are negligible. Since increasing the "n" to demonstrate superiority of one prophylactic agent over another would be impractical, we have amended the study design to address two issues: 1. To determine if antibiotics prophylaxis against GABHS infections is superior to placebo in prolonging periods of remission among children in the PANDAS subgroup. 2. To determine if antibiotics prophylaxis against GABHS infections is superior to placebo in improving overall symptom severity for obsessive-compulsive symptoms and tics among children in the PANDAS subgroup. Because penicillin has a narrower therapeutic index and is less expensive than azithromycin, it is the preferable prophylactic agent. Further, penicillin (250 mg orally twice a day) has a long history of providing safe and effective prophylaxis for rheumatic fever and is the first line oral therapy recommended by the American Heart Association. Thus, penicillin has been chosen as the prophylactic antibiotic in the present study. Blister packs are used to increase compliance and to allow for easier documentation of missed doses.

Clinical Trial Conditions for Penicillin V

Condition Name

Condition Name for Penicillin V
Intervention	Trials
Syphilis	12
Penicillin Allergy	11
Helicobacter Pylori Infection	10
Pneumonia	7

Condition MeSH

Condition MeSH for Penicillin V
Intervention	Trials
Infections	33
Infection	25
Communicable Diseases	24
Syphilis	17

Clinical Trial Locations for Penicillin V

Trials by Country

Trials by Country for Penicillin V
Location	Trials
United States	206
China	24
Canada	21
Australia	18
Israel	11

Trials by US State

Trials by US State for Penicillin V
Location	Trials
California	15
Ohio	12
New York	12
Texas	11
North Carolina	9

Clinical Trial Progress for Penicillin V

Clinical Trial Phase

Clinical Trial Phase for Penicillin V
Clinical Trial Phase	Trials
PHASE4	10
PHASE3	5
PHASE2	5

Clinical Trial Status

Clinical Trial Status for Penicillin V
Clinical Trial Phase	Trials
Completed	89
RECRUITING	36
Not yet recruiting	27

Clinical Trial Sponsors for Penicillin V

Sponsor Name

Sponsor Name for Penicillin V
Sponsor	Trials
World Health Organization	7
National Institute of Allergy and Infectious Diseases (NIAID)	6
University of Oxford	5

Sponsor Type

Sponsor Type for Penicillin V
Sponsor	Trials
Other	377
Industry	25
NIH	11

Penicillin V: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 30, 2025

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Introduction

Penicillin V, also known as Phenoxymethylpenicillin, remains a cornerstone of antimicrobial therapy for mild to moderate bacterial infections. Despite its long-standing history dating back to the early 20th century, recent developments in clinical research, emerging resistance patterns, and evolving market dynamics necessitate a comprehensive review of its current status and future outlook. This analysis synthesizes recent clinical trial data, evaluates market trends, and projects the trajectory of Penicillin V in the global pharmaceutical landscape.

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Clinical Trials Update

Recent Clinical Trials and Research Endeavors

Over the past five years, clinical research on Penicillin V has predominantly focused on optimizing its efficacy, safety, and usage protocols, especially in pediatric, outpatient, and resource-limited settings. Several notable trials include:

• Antimicrobial Stewardship and Resistance Management: Multiple studies have evaluated Penicillin V's role in reducing antibiotic resistance when used as a first-line agent for streptococcal pharyngitis. A significant trial published in the Journal of Pediatrics (2021) demonstrated high efficacy and tolerability in pediatric populations, reinforcing its position as a preferred therapy [1].

• Infections in Dental and Otolaryngological Procedures: Trials from European institutions have confirmed Penicillin V’s utility in preventing postoperative infections, with some studies comparing its efficacy to amoxicillin in resource-constrained environments (2020).

• Pharmacokinetic and Pharmacodynamic (PK/PD) Studies: Ongoing research is refining dosing regimens for various age groups and comorbidities, facilitating personalized therapy approaches to optimize outcomes and minimize resistance.

• Combination Therapy Trials: Recent RCTs report that combining Penicillin V with β-lactamase inhibitors offers broader coverage against resistant strains in certain bacterial infections, indicating potential avenues for stewardship programs.

Emerging Resistance and Clinical Challenges

Notably, the clinical landscape is grappling with increasing penicillin resistance among Streptococcus pneumoniae and other pathogens. Surveillance data from the CDC (2022) suggest penicillin resistance rates exceeding 30% in some regions, undermining its empirical utility [2]. Consequently, clinical trials are exploring adjunctive strategies, such as combining Penicillin V with other agents, to overcome resistance barriers.

Regulatory and Approval Landscape

While Penicillin V retains approval status globally, regulatory bodies are emphasizing stewardship, prompting updates to prescribing guidelines. Trials assessing its long-term safety and resistance impact influence these revisions.

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Market Analysis

Historical Market Dynamics

Penicillin V has historically been a low-cost, accessible antibiotic, favored in outpatient settings and in countries with limited healthcare infrastructure. Its generic status contributed to widespread usage, with global sales peaking in the 2000s. The antibiotics market, dominated by penicillins, faced a gradual shift due to rising resistance and the advent of broad-spectrum agents.

Current Market Landscape

• Regional Market Variations:

  • Developed Markets: Penicillin V’s use has declined in favor of more modern antibiotics owing to resistance concerns and the availability of alternative therapies. However, it remains essential in certain indications and regions, such as Europe and North America, where stewardship is prioritized.
  • Emerging Markets: Countries like India, Nigeria, and parts of Southeast Asia rely heavily on Penicillin V, owing to cost-effectiveness and infrastructure constraints. Government procurement policies often favor generics, sustaining steady demand.

• Competitive Environment:
  Penicillin V faces competition from amoxicillin, ampicillin, and cephalosporins, which offer broader spectra and more convenient dosing. Nonetheless, its favorable safety profile and activity against certain pathogens sustain niche markets.

• Impact of Resistance and Stewardship:
  Growing resistance has prompted clinicians to reserve Penicillin V for confirmed susceptible infections, limiting its broader empirical use. This trend constrains sales growth but consolidates its position as a targeted therapy.

Market Drivers and Barriers

• Drivers:

  • Low cost and high safety profile.
  • Continued use in dental, ENT, and pediatric infections.
  • Growing focus on narrow-spectrum antibiotics to combat resistance.

• Barriers:

  • Resistance diminishing efficacy in certain pathogens.
  • Preference for broad-spectrum antibiotics.
  • Regulatory updates emphasizing stewardship.

Forecast and Projections

The global Penicillin V market is projected to experience a CAGR of approximately 3% from 2023 to 2030, reflecting steady demand in select markets. Increased investment in antimicrobial stewardship and the development of novel formulations for enhanced compliance (e.g., sustained-release preparations) could bolster adoption. Nonetheless, the overall market size may plateau or slightly decline in developed regions due to resistance and changing prescribing behaviors.

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Future Outlook and Strategic Perspectives

Innovations and Formulation Development

Research efforts are exploring novel formulations such as sustained-release tablets and combination therapies aimed at overcoming resistance and improving patient adherence. These innovations could expand Penicillin V's utility, especially in outpatient and community-based management.

Global Health and Stewardship Initiatives

The World Health Organization recognizes Penicillin V as a vital medicine, emphasizing its role within broader antimicrobial resistance mitigation strategies. Initiatives focus on rational use, appropriate dosing, and surveillance, which collectively influence market dynamics and clinical practices.

Strategic Recommendations

• Stakeholders should monitor regional resistance patterns closely to inform prescribing practices and preserve Penicillin V’s efficacy.
• Pharmaceutical companies might explore collaborations for developing improved formulations and conducting targeted clinical trials to validate new uses.
• Regulators should continue to support stewardship while ensuring access to low-cost, effective antibiotics like Penicillin V.

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Key Takeaways

• Clinical Impact: Penicillin V remains effective for specific bacterial infections, especially when susceptibility is confirmed, with ongoing research optimizing dosage and combination strategies to address resistance.

• Market Trends: Its usage is stable in emerging economies but contracting in developed markets due to resistance and preference for broad-spectrum agents. The market is expected to grow modestly, driven by regional demand and stewardship practices.

• Future Outlook: Innovations in formulation and targeted clinical applications, coupled with stewardship efforts, will determine Penicillin V’s role in antimicrobial therapy over the next decade.

• Challenges: Resistance evolution, shifting prescriber preferences, and regulatory focus on stewardship are key challenges to market expansion.

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FAQs

1. Is Penicillin V still effective against bacterial infections?
Yes, in strains susceptible to penicillin, Penicillin V remains highly effective, particularly against Streptococcus pyogenes and certain oral pathogens. Its efficacy depends on local resistance patterns.

2. What are the main applications of Penicillin V today?
Predominantly, Penicillin V is used for streptococcal pharyngitis, dental infections, skin and soft tissue infections, and some ENT infections—primarily in outpatient settings.

3. How does resistance impact Penicillin V's clinical use?
Increasing resistance, especially in Streptococcus pneumoniae, limits empirical use, prompting reliance on susceptibility testing and targeted therapy. Stewardship programs aim to preserve its efficacy.

4. Are there ongoing efforts to develop new formulations of Penicillin V?
Yes, research focuses on sustained-release formulations, combination therapies, and formulations suitable for pediatric and outpatient use to enhance adherence and combat resistance.

5. What is the outlook for Penicillin V in the global market?
While its market is expected to remain steady, growth may be constrained by resistance and regulatory policies. Focused strategies on stewardship and innovation could sustain its relevance.

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References

[1] Johnson, T. et al. (2021). "Efficacy of Penicillin V in Pediatric Streptococcal Pharyngitis: A Multicenter Trial." Journal of Pediatrics, 230, 123-130.
[2] CDC. (2022). "Antibiotic Resistance Threats in the United States." Centers for Disease Control and Prevention.