Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 49 |
| INN | Gemtuzumab ozogamicin |
| Status | Approved |
| Drug code(s) | CMA-676, CDP-771 |
| Brand name | Mylotarg |
| mAb sequence source | mAb humanized |
| General Molecular Category | ADC |
| Format, general category | Full length Ab conjugate |
| Format details | None |
| Isotype (Fc) | IgG4 |
| Light chain isotype | kappa |
| Linker | AcBut acyl hydrazone-disulfide, Cleavable linker |
| Ave. DAR | 2 to 3 drugs per mAb |
| Conjugated/fused moiety | DNA binding, Calicheamicin |
| Discovery method/technology | None |
| Target(s) | CD33 |
| Indications of clinical studies | Acute myeloid leukemia |
| Primary therapeutic area | Cancer |
| Most advanced stage of development (global) | Approved EU, US, Japan, Australia |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | May 15, 1995 |
| Start of Phase 2 | May 15, 1997 |
| Start of Phase 3 | |
| Date BLA/NDA submitted to FDA | October 29, 1999 |
| Year of first approval (global) | 2000 |
| Date of first US approval | May 17, 2000 |
| INN, US product name | Gemtuzumab ozogamicin |
| US or EU approved indications | Acute myeloid leukemia (adults with newly diagnosed CD33-positive AML, and adults and children 2 years and older with relapsed or refractory CD33-positive AML) |
| Company | UCB |
| Licensee/Partner | Pfizer |
| Comments about company or candidate | Approved in US on Sep 1, 2017; Approved 5/17/2000 in US (withdrawn in 2010); Approved in the EU on April 19, 2018. Feb 2018: EMA issued a positive opinion; EC decision is pending. 2000 (withdrawn US and EU in 2010); approved again in US in 2017 and EU in 2018 July 2017: FDA's Oncologic Drugs Advisory Committee voted 6-1 in favor of putting Mylotarg, or gemtuzumab ozogamicin, back on the market. Mylotarg is indicated in combination with chemotherapy agents for adult patients with acute myeloid leukemia who have not undergone other treatments. Mylotarg (gemtuzumab ozogamicin) -- A Biologics License Application (BLA) for Mylotarg was accepted for filing by the FDA in January 2017 and a Marketing Authorization Application (MAA) was validated for review by the European Medicines Agency (EMA) in December 2016. Mylotarg is being evaluated for the potential treatment of adult patients with acute myeloid leukemia (AML). Mylotarg was originally approved under the FDA’s accelerated approval program in 2000 for use as a single agent in first relapse patients with CD33-positive AML who were 60 years or older. In 2010, Pfizer voluntarily withdrew Mylotarg after a confirmatory Phase 3 trial did not show a clinical benefit and the fatal induction toxicity rate was significantly higher in the Mylotarg arm. The recent regulatory submissions are based on additional data from a Phase 3 randomized, open-label study (ALFA-0701) that evaluated the addition of Mylotarg to standard induction chemotherapy using an alternative fractionated dosing schedule in 280 adult, de novo, AML patients aged 50-70 years old, as well as a meta-analysis of patient-level data from over 3,000 patients in five randomized Phase 3 studies (including ALFA-0701) spanning 10 years of research. The PDUFA goal date for a decision by the FDA is in September 2017. Mylotarg originates from a collaboration between Pfizer and Celltech, now UCB. Pfizer has sole responsibility for all manufacturing and clinical development activities for this molecule. |
| Full address of company | Allée de la Recherche, 60 1070 Brussels Belgium Europe Belgium https://www.ucb.com/contact |
Humanized anti-CD33 IgG4; ADC. S228P hinge mutation. acid-labile 4-(4′-acetylphenoxy) butanoic acid (acetyl butyrate) linker
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |