Pfizer's leukaemia drug gets NHS funding in England and Wales

Pfizer’s Mylotarg has been approved by the English and Scottish NHS cost watchdogs as a restricted use treatment for a certain type of acute myeloid leukaemia (AML).

Mylotarg (gemtuzumab ozogamicin) is approved for NHS use in combination with chemotherapy for previously untreated, newly diagnosed, CD33-positive AML.

It was previously only approved to treat patients whose cytogenetics showed the CD-33 mutation, or where they were so far unknown, and could not be used where the presence of the CD-33 mutation was deemed unclear.

The National Institute of Health and Care Excellence (NICE) made its recommendation last week; the Scottish Medicines Consortium (SMC) followed suit this week.

NICE’s Final Appraisasl Determination (FAD) backed the drug to be used for patients aged 15-plus, in combination with daunorubicin and cytarabine for untreated CD33-positive AML.

A phase 3 clinical trial, ALFA-0701, showed that Mylotarg plus chemotherapy gave a statistically significant and meaningful improvement in median event-free survival, versus chemotherapy alone – 17.3 months compared with 9.5 months respectively.

It also demonstrated a statistically significant improvement in relapse-free survival, versus chemotherapy alone: 28.0 months and 11.4 months respectively.

AML is a rare, aggressive blood cancer that affects approximately 2,600 people in the UK each year. It has an average life expectancy of less than 10 months if untreated.

Professor Nigel Russell from the Centre for Clinical Haematology, Nottingham University Hospital Trust, said: “Over the past few decades, there has been little innovation in how we treat AML. 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.”

In May, NICE told Pfizer it had to cut the price of the drug to secure funding from the NHS. In draft guidance issued at the time, it said Mylotarg was too pricey for patients with AML unless it was clear that they had the CD-33 mutation.

Mylotarg was first approved in 2000 and was the world’s first antibody-drug conjugate, consisting of a cancer-seeking antibody bound to a cytotoxic compound unleashed when the drug binds to its target.