- Global Pharma News & Resources

Pfizer statement: NICE Recommends MYLOTARG™&#9660 (gemtuzumab ozogamicin) for Treatment of Previously Untreated, De Novo, CD33-positive Acute Myeloid Leukaemia (AML) in Combination with Chemotherapy

Craig Eagle, Head of Oncology, Pfizer UK said: “We welcome NICE’s final decision to recommend gemtuzumab ozogamicin for eligible patients living with AML. Pfizer has worked tirelessly with the AML community to ensure patients in the UK have access to appropriate treatment options and this important, potentially life-changing medicine offers renewed hope for some people living with AML in England and Wales.”


“Over the past few decades, there has been little innovation in how we treat AML,” said haematologist Professor Nigel Russell from the Centre for Clinical Haematology, Nottingham University Hospital Trust.  “Despite slow improvements in outcomes, the prognosis for patients with this type of blood cancer remains poor. Gemtuzumab ozogamicin is a targeted therapy that is given with standard chemotherapy and prolongs the time spent in remission compared to standard treatments. It provides a welcome addition to the treatment options for eligible patients with AML and is an important step towards ensuring that some very sick blood cancer patients can access appropriate treatments to achieve a prolonged, complete remission.”


About the gemtuzumab ozogamicin FAD announcement

  • Following today’s decision by the National Institute of Health and Care Excellence (NICE), gemtuzumab ozogamicin will be available through the National Health Service (NHS) for eligible patients in England and Wales.
  • The Final Appraisal Determination (FAD), published today by NICE, recommends the use of MYLOTARG™▼ (gemtuzumab ozogamicin) in combination with daunorubicin and cytarabine as an option for untreated CD33-positive acute myeloid leukaemia (AML), except acute promyelocytic leukaemia, in people 15 years and over, only if they start induction therapy when either the cytogenetic test confirms that the disease has favourable, intermediate or unknown cytogenetics (that is because the test was unsuccessful) or when their cytogenetic test results are not yet available, and they start consolidation therapy when their cytogenetic test confirms that the disease has favourable, intermediate or unknown cytogenetics (because the test was unsuccessful).[i]
  • AML is a rare and aggressive blood cancer that affects approximately 2,600 people in the UK each year.[ii] If left untreated, the average life expectancy is less than 10 months.[iii],[iv]
  • The goal of AML treatment is to help patients achieve a prolonged complete remission,[v] however, few new treatment options have become available for AML patients in the last few decades,[vi] with chemotherapy remaining the standard-of-care since the 1970s.[vii]
  • Gemtuzumab ozogamicin is a targeted agent that has been shown to be effective in treating newly diagnosed CD33-positive AML patients.[viii],[ix]
  • In the Phase 3 clinical trial, ALFA-0701, gemtuzumab ozogamicin in combination with chemotherapy offers a statistically significant and meaningful improvement in median event-free survival, versus chemotherapy alone (17.3 months and 9.5 months respectively (HR, 0.562 [95% CI: 0.415, 0.762], p=0.0002)ix9and a statistically significant improvement in relapse-free survival, versus chemotherapy alone (28.0 months and 11.4 months respectively (HR, 0.526 [95% CI: 0.362, 0.764], p=0.0006).viii,ix8,9
  • In ALFA-0701, 81% of patients treated with gemtuzumab ozogamicin reached remission, in comparison to 75% of those treated with the standard-of-care, but this difference was not statistically significant. viii 8
  • The overall safety profile of gemtuzumab ozogamicin is based on data from the combination therapy study ALFA-0701, monotherapy studies, and from post-marketing experience.iX9 Clinically relevant serious adverse reactions were hepatotoxicity, including veno-occlusive disease (VOD) /sinusoidal obstruction syndrome (3.8%), haemorrhage (9.9%), severe infection (41.2%), and tumour lysis syndrome (1.5%).ix9  The most common adverse reactions (> 30%) in the combination therapy study were haemorrhage and infection.ix9 The most frequent (≥ 1%) adverse reactions that led to permanent discontinuation in the ALFA-0701 study were thrombocytopenia, VOD, haemorrhage and infection.ix9


About gemtuzumab ozogamicin

  • Gemtuzumab ozogamicin is an antibody drug conjugate that consists of a monoclonal antibody connected to the anti-tumour agent calicheamicin. It works by targeting the antigen, CD33, which is expressed on the surface of myeloblasts found in up to 90 % of AML patients.x,xi,xii10,11,12
  • When gemtuzumab ozogamicin binds to the CD33 antigen on the cell surface, it is absorbed into the cell and calicheamicin is released causing cell death.xi,xii11,12
  • On 23rd April 2018, the European Commission (EC) granted marketing authorisation for gemtuzumab ozogamicin in combination with chemotherapy for the treatment of patients age 15 years and above with previously untreated, de novo, CD33-positive AML, except acute promyelocytic leukaemia (APL).xiii13


For further information, please contact:

Pfizer Press Office
Tel: 0845 300 8033

GCI Health Pfizer Team

Tel: 0207 072 4218


Editor Details

Last Updated: 08-Oct-2018