NEW oral treatment for AML patients approved in Scotland, England and Wales

Webpage last updated on: 9th May 2024

What’s the news?

Today, Leukaemia Care welcomes the decision by the National Institute for Healh and Care Excellence (NICE) to approve ivosidenib (Tibsovo©) with azacitidine for use on the NHS in Wales and England, with Northenr Ireland likely to follow this too. This is a treatment for patients with newly diagnosed acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy. It was approved for use in Scotland back in March 2024.

Leukaemia Care submitted a written response to NICE and the SMC representing the views of AML patients. A submission is created by drawing on experiences of individual patients and data from our surveys. We also nominated a patient, Esther Beswick, to speak about the experience of AML patients at the committee meeting for the NICE decision. Esther described problems with current treatments from the viewpoint of AML patients.

Our Policy and Evidence Manager, Charlotte Crowley, said:

“Today’s decision will provide access to the first treatment specifically targeted at AML patients with an IDH1 mutation who cannot undergo standard induction chemotherapy. This an important step forward for this group of patients who cannot undergo chemotherapy, that now have more treatment options available to them at the beginning of their AML journey. We are pleased that now this treatment will be provided across all the UK nations.”

Esther Beswick, AML patient, said of today’s NICE decision:

“I know from my own diagnosis and from volunteering with Leukaemia Care that AML continues to cost the lives of too many people. I’m pleased that the committee considered my comments on the need for more treatments and was able to approve this treatment today. Ivosidenib is especially important for those who cannot tolerate the existing treatment, many of which have been used for a long time. It is time for treatments to be kinder to patients as well as effective. ”

Why is this news important for AML patients with an IDH1 mutation?

There are currently no treatments that are specifically targeted for AML patient with an IDH1 mutation. Ivosidenib with azacitidine will be the first treatment specifically for this group of patients who cannot undergo standard induction chemotherapy.

In newly diagnosed AML, the preferred primary induction treatment is intensive chemotherapy. However, this treatment can be unsuitable for AML patients with an IDH1 mutation as they are usually older and have pre-existing medical conditions.

Ivosidenib with azacitidine has been shown to improve event-free survival, overall survival, and likelihood of complete remission in the target group than azacitidine alone.

This new treatment also has an improved side effect profile than azacitidine alone and some of the side effects can be managed by clinicians. Under existing treatments, patients potentially face the harmful side effects of systemic chemotherapy, which can be avoided with an oral targeted treatment such as ivosidenib with azacitidine.

There is also the benefit of ivosidenib being an oral treatment. This can be more convenient to patients as it can be taken at home. This can help reduce travel time and the financial burden associated with hospital travel. This can also allow patients to spend more time with friends and family outside a hospital setting.

How is the treatment administered?

Ivosidenib is an oral tablet treatment that is taken once every day (2X 250mg tablets). It is taken for as long as a clinical benefit is observed, or until it no longer tolerated by the patient.

Azacitidine is administered via an intravenous line. This is usually administered in a hospital or medical facility, or at home for patients unable to travel for health reasons.

More information on AML

Download acute myeloid leukaemia (AML) booklet