NICE is recommending Mylotarg for previously untreated, de novo, CD33-positive acute myeloid leukaemia in combination with chemotherapy.

The Final Appraisal Determination now backs use of the drug in combination with daunorubicin and cytarabine for untreated CD33-positive AML (except acute promyelocytic leukaemia) in people 15 years and over with favourable, intermediate or unknown cytogenetics (because the test was unsuccessful).

Earlier draft guidelines only recommended Mylotarg (gemtuzumab ozogamicin) for patients with favourable or unknown cytogenetics, but not for those with intermediate cytogenetics, which would have excluded around 65 percent of the eligible patient population.

Mylotarg is a targeted therapy consisting of an antibody connected to an anti-tumor agent that is toxic to cells, and thought to work by taking the anti-tumour agent to the AML cells that express the CD33 antigen, blocking the growth of cancerous cells and causing cell death.

The treatment won European approval in May this year after clinical trials showed 81 percent of patients taking the drug reached remission, and that Mylotarg 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).

“We welcome NICE’s final decision to recommend gemtuzumab ozogamicin for eligible patients living with AML,” said Craig Eagle, head of Oncology, Pfizer UK.

“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,” noted 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.”