Daraprim

(Pyrimethamine)

ApprovedSmall molecule

FFondazione Matilde Tettamanti Menotti De Marchi Onlus

Pyrimethamine is used to treat malaria and certain parasitic infections like toxoplasmosis. It works by stopping parasites from growing and multiplying in the body.[2] Daraprim is currently approved and in use across 24 indications[4].[1]

Daraprim is one of 509 small molecule programs targeting HIV infection. It is currently in Approved.

Indications

Total Trials

Active Trials

508

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[4][1]

Indication

HIV InfectionApproved

ToxoplasmosisActive, not recruiting

Depressive DisorderApproved

GM2 GangliosidosisActive, not recruiting

PneumocystosisActive, not recruiting

Head and Neck Malignant NeoplasiaApproved

SchistosomiasisApproved

Plasmodium Falciparum MalariaApproved

Congenital ToxoplasmosisApproved

Cerebral ToxoplasmosisActive, not recruiting

HIV InfectionApproved

Approved

ToxoplasmosisApproved

Active, not recruiting

Depressive DisorderApproved

Approved

GM2 GangliosidosisPhase 1

Active, not recruiting

PneumocystosisApproved

Active, not recruiting

Head and Neck Malignant NeoplasiaApproved

Approved

SchistosomiasisApproved

Approved

Plasmodium Falciparum MalariaApproved

Approved

Congenital ToxoplasmosisApproved

Approved

Cerebral ToxoplasmosisPhase 2

Active, not recruiting

Showing 10 of 24 indications

Competition

Competitive Landscape

583 competitors in Schizophrenia

#DrugCompanyPhaseTrials

Daraprim

FFondazione Matilde Tettamanti Menotti De Marchi Onlus

Approved

1 2

Celexa

AbbVie

Preclinical

4 3

Zocor

OOrganon and Co

Preclinical

6 4

Ibrance

Depakote

Comtan

Orion Corporation, Orion Pharma

Preclinical

1 7

Rapamune

PPrism Pharmaceuticals

Preclinical

1 8

Inversine

TTargacept Inc.

Preclinical

1 9

Persantine

Boehringer Ingelheim

Preclinical

1 10

Permax

VValeant Pharmaceuticals

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2028

Phase 1/2Not yet recruiting

HIV

NCT07384624

Q4 2030

Post-ApprovalRecruiting

Congenital Toxoplasmosis

NCT00004317

Clinical

Trials by Indication[1]

Encephalitis4 trials

Toxoplasmic Encephalitis in Patients With AIDS. Treatment and Prevention of Relapse

Completed·PPalo Alto Medical Foundation·N/A·NCT00002064

A Pilot Study of Oral Clindamycin and Pyrimethamine for the Treatment of Toxoplasmic Encephalitis in Patients With AIDS

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·N/A·NCT00000674·30 enrolled

Phase II Randomized Open-Label Trial of Atovaquone Plus Pyrimethamine and Atovaquone Plus Sulfadiazine for the Treatment of Acute Toxoplasmic Encephalitis

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00000794·100 enrolled

Show 1 more trial

A Randomized Prospective Study of Pyrimethamine Therapy for Prevention of Toxoplasmic Encephalitis in HIV-Infected Individuals With Serologic Evidence of Latent Toxoplasma Gondii Infection

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·N/A·NCT00000666·600 enrolled

HIV2 trials

LRAs United as a Novel Anti-HIV Strategy.

Completed·EErasmus Medical Center·Phase 1/2·NCT03525730·28 enrolled

Combining Latency Reversing Agents to Address the HIV Reservoir

Not yet recruiting·EErasmus Medical Center·Phase 1/2·NCT07384624·30 enrolled

Sandhoff disease1 trial

Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

Completed·TThe Hospital for Sick Children·Phase 1/2·NCT01102686·20 enrolled

Vaccination1 trial

Sanaria PfSPZ Challenge With Pyrimethamine or Chloroquine Chemoprophylaxis Vaccination (PfSPZ-CVac Approach): A Randomized Double Blind Placebo Controlled Phase I/II Trial to Determine Safety and Protective Efficacy Against Natural Plasmodium Falcipa...

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT03952650·252 enrolled

Cerebral Toxoplasmosis1 trial

Primary Prophylaxis of Cerebral Toxoplasmosis in HIV-Infected Patients

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00000643·150 enrolled

15 more indications

Completed·WWeill Medical College of Cornell University·Phase 1/2·NCT01083667·32 enrolled

GM2 gangliosidosis1 trial

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

Withdrawn·EExsar Corporation·Phase 1·NCT00679744

Azithromycin1 trial

A Study of Azithromycin Plus Pyrimethamine in the Treatment of a Brain Infection in Patients With AIDS

Completed· Pfizer logo

Pfizer·Phase 1·NCT00000966·45 enrolled

Drug1 trial

An Investigational Study to Determine the Drug Level Profile of BMS-986165 in Healthy Male Volunteers Following Transporter Inhibition.

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 1·NCT04086719·16 enrolled

Myelodysplastic Syndromes1 trial

Pyrimethamine for Intermediate/High-risk Myelodysplastic Syndromes (MDS)

Withdrawn·MMontefiore Medical Center·Phase 1·NCT03057990

Schizophrenia1 trial

Effect of Specific Anti-Toxoplasmatic Add-on Medication in Toxoplasma Gondii Seropositive Individuals With Schizophrenia or Major Depression

Completed·ZZentrum für Integrative Psychiatrie·Phase 3·NCT00300404·40 enrolled

Chronic Lymphocytic Leukemia/small Lymphocytic Lymphoma1 trial

Pyrimethamine for the Treatment of Relapsed Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

Completed·—·Phase 1/2·NCT01066663·20 enrolled

Congenital Toxoplasmosis1 trial

Pyrimethamine, Sulfadiazine, and Leucovorin in Treating Patients With Congenital Toxoplasmosis

Recruiting·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 4·NCT00004317·600 enrolled

Head and Neck Squamous Cell Carcinoma1 trial

Pyrimethamine as an Inhibitor of NRF2 in HPV-unrelated Locally Advanced Head and Neck Squamous Cell Carcinoma

Completed·WWashington University School of Medicine·Phase 1·NCT05678348·22 enrolled

Prophylaxis1 trial

Sanaria PfSPZ Challenge With Pyrimethamine Chemoprophylaxis (PfSPZ-CVac Approach): Phase 1 Trial to Determine Safety and Protective Efficacy of Sanaria PfSPZ Challenge With Concurrent Pyrimethamine Treatment That Inhibits Development of Asexual B...

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT02511054·57 enrolled

Combination Therapy1 trial

Optimizing Reversal of HIV Latency With Combination Therapy

Unknown status·EErasmus Medical Center·Phase 1/2·NCT06240520·49 enrolled

Unspecified2 trials

Dose Escalation PfSPZ-CVac

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT03083847·55 enrolled

Phase I Study of Pyrimethamine in Healthy Japanese and Caucasian Subjects

Completed· GSK logo

GSK·Phase 1·NCT03258762·14 enrolled

Regulatory

FDA Approval Timeline[5]

Jan 1, 1982

DARAPRIM

NDA NDA008578

Sources

References

[1]
ClinicalTrials.gov — 25 trial records for Pyrimethamine. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Daraprim drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Pyrimethamine compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Pyrimethamine. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA008578). U.S. Food & Drug Administration.
[6]
SEC EDGAR — Fondazione Matilde Tettamanti Menotti De Marchi Onlus company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Dihydrofolate Reductase Inhibitor[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: FFondazione Matilde Tettamanti Menotti De Marchi Onlus

Daraprim

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q3 2028

Q4 2030

Trials by Indication[1]

Encephalitis4 trials

HIV2 trials

Sandhoff disease1 trial

Vaccination1 trial

Cerebral Toxoplasmosis1 trial

Pneumonia1 trial

Lymphoproliferative Syndrome1 trial

Treatment1 trial

Familial Amyotrophic Lateral Sclerosis1 trial

GM2 gangliosidosis1 trial

Azithromycin1 trial

Drug1 trial

Myelodysplastic Syndromes1 trial

Schizophrenia1 trial

Chronic Lymphocytic Leukemia/small Lymphocytic Lymphoma1 trial

Congenital Toxoplasmosis1 trial

Head and Neck Squamous Cell Carcinoma1 trial

Prophylaxis1 trial

Combination Therapy1 trial

Unspecified2 trials

FDA Approval Timeline[5]

References

Drug Info

Quick Facts

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