AML is a type of blood cancer, which progresses quickly and aggressively, and requires immediate treatment. Each year in the UK around 3,100 people are diagnosed with AML, which can affect the different types of blood cells in the bone marrow, and can lead to bleeding, anaemia and infections.
Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said:
Our key priorities are keeping patients safe and enabling their access to high quality, safe and effective medical products.
We're assured that the appropriate regulatory standards for the approval of this medicine have been met.
As with all medicines, we will keep its safety under close review.
Quizartinib has been approved to be used alongside chemotherapy as first line treatment for adults who have AML with a mutation in the FLT3 gene called 'FLT3-ITD'.
Treatment is taken orally, once a day at around the same time for two weeks, during each cycle of chemotherapy. The prescribing doctor will determine the dose of quizartinib that should be taken and for how long. Following completion of chemotherapy, the doctor may continue the patient's treatment with quizartinib.
Quizartinib blocks the action of tyrosine kinases, the proteins responsible for the body producing large numbers of abnormal bone marrow cells that do not mature to become healthy cells.
By slowing down or stopping the abnormal cells from dividing and growing uncontrollably, quizartinib helps immature cells grow into normal cells.
This regulatory approval is supported by evidence from a randomised, double-blind, placebo-controlled, phase 3 clinical trial involving 539 adult patients (aged between 18 and 75 years) newly diagnosed with FLT3-ITD positive AML.
Patients were randomised to receive either quizartinib (268 patients) or a placebo (271 patients) once daily for two weeks in each 28-day treatment cycle in combination with standard chemotherapy, followed by either quizartinib or placebo once daily for up to 36 cycles of 28 days.
The study demonstrated a statistically significant improvement in overall survival (time from randomisation to death from any cause) for patients receiving quizartinib.
Patients receiving quizartinib had a median overall survival of 31.9 months compared to 15.1 months for those receiving the placebo.
The most common side effects of the medicine (which may affect more than 1 in 10 people) include abnormal liver enzymes, low levels of blood platelets, anaemia, low levels of certain white blood cells, diarrhoea, nausea, stomach pain, headache, vomiting, swelling (face, arms and legs), nose and throat infection, decreased appetite, severe nosebleeds, fungal infections, herpes infections, indigestion and bacteria in the blood.
As with any medicine, the MHRA will keep the safety and effectiveness of quizartinib under close review. Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the MHRA Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.
Notes
- The new marketing authorisation was granted on 8 March 2024 to Daiichi Sankyo UK Ltd.
