The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation to Daiichi Sankyo for quizartinib (Vanflyta) for use as a first-line treatment in adults with acute myeloid leukaemia (AML) with a FLT3-ITD mutation.
The decision allows for the oral administration of quizartinib in combination with chemotherapy - a significant development in the treatment of AML.
Taken once daily, quizartinib targets tyrosine kinases to hinder the growth of abnormal bone marrow cells.
The treatment duration and dosage are determined by the prescribing physician and may continue post-chemotherapy.
The approval is backed by a double-blind, randomised, placebo-controlled Phase III clinical trial in 539 adult subjects aged between 18 and 75 years with newly diagnosed FLT3-ITD positive AML.
The study demonstrated a statistically significant improvement in overall survival (time from randomisation to death from any cause) for patients who received quizartinib.
Patients receiving quizartinib had a median overall survival of 31.9 months compared to 15.1 months for those receiving a placebo.
Abnormal liver enzymes, low blood platelet and white blood cell levels, anaemia, diarrhoea, nausea, stomach pain and headache were the most common side effects linked to the trial.
MHRA Healthcare Quality and Access interim executive director Julian Beach stated: “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.”
In another development, the MHRA also gave marketing authorisation to Pfizer for etrasimod (Velsipity) for the treatment of moderately to severely active ulcerative colitis.
Etrasimod is prescribed as a long-term treatment and should be initiated only under a doctor's supervision.
The approval is supported by two clinical studies, ELEVATE UC 52 and ELEVATE UC 12, indicating its efficacy for patients where standard treatments were inadequate.
The UK National Institute for Health and Care Excellence (NICE) has also published a final guidance recommending etrasimod for ulcerative colitis.
With the NICE recommendation, more than 25,000 people in England will be eligible to receive the treatment.
The latest development comes after the MHRA approved Marinus Pharmaceuticals’ ganaxolone, marketed as Ztalmy, an anti-seizure medication for cyclin-dependent kinase-like 5 (CDLK5) deficiency disorder.
