NEW oral treatment for AML patients approved in Scotland

Leukaemia Care are pleased to announce that newly diagnosed acute myeloid leukaemia (AML) patient with an IDH1 mutation who are ineligible for standard induction chemotherapy now have access to ivosidenib with azacitidine thanks to the decision made by the Scottish Medicines Consortium (SMC) today.

Webpage last updated on: 11th March 2024

What is the news?

Today, Leukaemia Care welcomes the decision by the Scottish Medicines Consortium (SMC) to approve ivosidenib (Tibsovo©) with azacitidine for use on the NHS in Scotland. 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.

Leukaemia Care submitted a written response to the SMC representing the views of AML patients. A submission is created by drawing on experiences and data, and advocating for equity of access for Scottish patients.

This treatment is currently going through The National Institute for Health and Care Excellence’s (NICE) process in England which Leukaemia Care are currently involved in. We expect NICE to make a decision in the following months and will update this blog when we have more information.

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 hope to see NICE approve ivosidenib as well in order to ensure equity of access across all UK nations.”

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.

How does this decision affect access to treatments elsewhere in the UK?

Decisions made by the Scottish Medicines Consortium (SMC), such as today’s decision, only apply to patients in Scotland.

England has a separate body that reviews treatments for use in NHS England, the National Institute for Health and Care Excellence’s (NICE). Wales and Northern Ireland usually choose to adopt NICE’s recommendations. The manufacturer must submit separately, and a separate process followed before treatments are made available there. We will continue to work with NICE and companies to ensure everyone across the UK has equal access to effective treatments. If approved in England, Wales, and Northern Ireland we will update you with another news article.

Do you need support?

If you want to know more about what treatments are available for you, how treatment decisions are made or have any other question related to treatment, please contact us via our helpline 08088 010 444,  or send your question to WhatsApp on 07500 068 065 (services available Monday to Friday 9am – 5pm). Alternatively you can email our team at

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