Gemtuzumab ozogamicin

Overview

Gemtuzumab ozogamicin is a recombinant humanized IgG4 kappa antibody which is conjugated with calicheamicin derivative, a cytotoxic antitumor antibiotic isolated from fermentation of Micromonospora echinospora ssp. calichensis. Gemtuzumab ozogamicin has approximately 50% of the antibody loaded with 4-6 moles calicheamicin per mole of antibody . The antibody is specifically directed against the CD33 antigen present on leukemic myeloblasts in most patients with acute myeloid leukemia (AML). By binding to the CD33 antigen on tumors, the cytotoxic agent blocks the growth of cancerous cells and causes cell death. Marketing approval of gemtuzumab ozogamicin was granted on May 17, 2000 by FDA as a treatment for patients with CD33-positive AML in first relapse who are 60 years of age or older and who are not considered candidates for cytotoxic chemotherapy . However, it was voluntarily withdrawn from the market in 2010 due to safety concerns, increased patient deaths and insufficient evidence of clinical benefit during confirmatory trials . On September 1 2017, gemtuzumab ozogamicin was again approved for the treatment of adults with newly diagnosed CD33-positive acute myeloid leukemia but with a lower dosing regimen and a different schedule in combination with chemotherapy or on its own . It is also indicated for the treatment of patients aged 2 years and older with CD33-positive AML who have experienced a relapse or who have not responded to initial treatment (refractory) .

Indication

Indicated for the treatment of patients with CD33 positive acute myeloid leukemia in first relapse who are 60 years of age or older and who are not considered candidates for other cytotoxic chemotherapy. Indicated for the treatment of patients aged 2 years and older with CD33-positive AML who have experienced a relapse or who have not responded to initial treatment (refractory).

Associated Conditions

Acute Myeloid Leukemia
Refractory Acute Myeloid Leukemia (AML)
Relapsed Acute Myelogenous Leukemia (AML)

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Testing the Addition of Venetoclax or Gemtuzumab Ozogamicin (GO) to Usual Treatment Regimen (Cytarabine and Daunorubicin, "7+3") for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) to Improve Response (A MyeloMATCH Treatment Trial)	2025/04/09	NCT06917911	Phase 2	Not yet recruiting	National Cancer Institute (NCI)
A Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia	2023/07/21	NCT05955261	Phase 2	Recruiting	St. Jude Children's Research Hospital
Genetic Ablation of CD33 in HSC to Broaden the Therapeutic Index of CD33-directed Immunotherapy in Patients with AML	2022/12/23	NCT05662904	Phase 1	Not yet recruiting	German Cancer Research Center
Vyxeos for Induction of Low- or Intermediate-risk.	2022/10/31	NCT05599360	Phase 2	Active, not recruiting	Shaare Zedek Medical Center
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)	2022/10/03	NCT05564390	Phase 2	Recruiting	National Cancer Institute (NCI)
CPX-351 in Combination With Gemtuzumab Ozogamicin in Newly Diagnosed AML	2022/09/28	NCT05558124	Phase 1	Recruiting	H. Lee Moffitt Cancer Center and Research Institute
Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)	2022/01/10	NCT05183035	Phase 3	Recruiting	LLS PedAL Initiative, LLC
Liposomal Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin for the Treatment of Relapsed Refractory Pediatric Patients With Acute Myeloid Leukemia	2021/06/07	NCT04915612	Phase 1	Completed	M.D. Anderson Cancer Center
Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS	2021/04/19	NCT04849910	Phase 1	Active, not recruiting	Vor Biopharma
Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia	2021/03/11	NCT04793919	Phase 2	Recruiting	Associazione Italiana Ematologia Oncologia Pediatrica

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
Mylotarg	Wyeth Pharmaceuticals LLC, a subsidiary of Pfizer Inc.	0008-4510	INTRAVENOUS	5 mg in 5 mL	9/3/2021

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Mylotarg	Pfizer Europe MA EEIG,Boulevard de la Plaine 17,1050 Bruxelles,Belgium	Authorised	4/19/2018

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
MYLOTARG POWDER FOR CONCENTRATE FOR SOLUTION FOR INFUSION 5MG/VIAL	Wyeth Pharmaceutical Division of Wyeth Holdings LLC	SIN16243P	POWDER, FOR SOLUTION	5.0 mg/vial	6/21/2021

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
MYLOTARG gemtuzumab ozogamicin 5 mg powder for injection vial	316671	Pfizer Australia Pty Ltd	Medicine	A	4/9/2020

Related News

ADC Market in NSCLC Projected to Reach $3.9 Billion by 2032, Led by AstraZeneca's Datroway

2 months ago

The antibody-drug conjugate (ADC) market in non-small-cell lung cancer is projected to grow at a 6.8% CAGR across seven major markets and exceed $3.9 billion by 2032.

Antibody-Drug Conjugates (ADCs) Emerge as Promising Therapeutic Modality in Oncology

6 months ago

Antibody-drug conjugates (ADCs) combine monoclonal antibody specificity with potent small molecule cytotoxicity, precisely delivering toxins to tumors and sparing normal tissues.

Vor Bio Secures $55.6M Private Placement to Advance Blood Cancer Cell Therapy Pipeline

7 months ago

Vor Bio has secured a $55.6 million private placement led by Reid Hoffman, with participation from RA Capital Management. The funding will support the development of their cell therapy programs, including trem-cel for acute myeloid leukemia, with key clinical data readouts expected in 2025.

FDA Clears IND for SystImmune's BL-M11D1 in Relapsed/Refractory AML

9 months ago

• The FDA has cleared SystImmune's Investigational New Drug (IND) application for BL-M11D1, an antibody-drug conjugate (ADC), for relapsed/refractory Acute Myeloid Leukemia (AML). • BL-M11D1 targets CD33, a protein expressed on myeloid cells, and is designed to trigger antibody-dependent cellular cytotoxicity (ADCC) and induce tumor cell death. • A Phase 1 clinical trial, BLM11D1-HM-101, is set to begin in the United States to assess the safety, tolerability, pharmacokinetics, and preliminary efficacy of BL-M11D1. • BL-M11D1 comprises a monoclonal antibody binding CD33 and a topoisomerase I inhibitor payload, potentially offering a novel therapeutic option for AML patients.

Vor Biopharma Announces Positive Data and New CD45 ADC Program, Analysts See Significant Upside

10 months ago

• Vor Biopharma reported updated Phase 1/2 VBP101 study results showing promising relapse-free survival data in AML/MDS patients, exceeding historical benchmarks. • Initial PK data for VCAR33ALLO demonstrated in vivo expansion at the lowest dose, with dose escalation planned, indicating potential efficacy. • The company is developing a new CD45 ADC program (VADC45) for conditioning regimens, offering a fast path to commercial development and partnership opportunities. • Analysts maintain an Outperform rating with a price target of US$11.00, citing significant upside potential from trem-cel and CD45-based depletion approaches.

Help Us Improve

Your feedback helps us provide better drug information and insights.

AI-Powered Research

Premium Access