Carbaglu

(Carglumic Acid)

Phase 3Small molecule

RRECORDATI GROUP

Carbaglu is one of 4 small molecule programs targeting Propionic Acidemia. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

Propionic AcidemiaActive, not recruiting

Propionic AcidemiaPhase 3

Active, not recruiting

Competition

Competitive Landscape

10 competitors in Propionic Acidemia

#DrugCompanyPhaseTrials

Carbaglu

RRECORDATI GROUP

Phase 3

1 2

Glutamine

Sanofi

Phase 1

1 3

Mrna-3927

Moderna

Phase 2

1 4

N-Carbamylglutamate

MMendel Tuchman

Phase 2

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

Timeline

Expected Milestones

Q3 2029

Recruiting

CAR

NCT04176523

Q2 2032

Recruiting

Observational Study

NCT05040178

Clinical

Trials by Indication[1]

Propionic Acidemia1 trial

Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

Completed·KKing Abdullah International Medical Research Center·Phase 3·NCT02426775·33 enrolled

CAR1 trial

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

Recruiting·RRECORDATI GROUP··NCT04176523·95 enrolled

Observational Study1 trial

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

Recruiting·RRECORDATI GROUP··NCT05040178·20 enrolled

Efficacy1 trial

Long-term Efficacy of Carglumic Acid in Organic Acidemia.

Completed·NNational Taiwan University Hospital·N/A·NCT04284917·3 enrolled

Regulatory

FDA Approval Timeline[4]

Mar 18, 2010

CARBAGLU

NDA NDA022562

Sources

References

[1]
ClinicalTrials.gov — 4 trial records for Carglumic Acid. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Carglumic Acid compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Carglumic Acid. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA022562). U.S. Food & Drug Administration.
[5]
SEC EDGAR — RECORDATI GROUP company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: RRECORDATI GROUP

Carbaglu

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2029

Q2 2032

Trials by Indication[1]

Propionic Acidemia1 trial

CAR1 trial

Observational Study1 trial

Efficacy1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

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