NICE approves midostaurin for treating Acute Myeloid Leukaemia (AML)

NICE have issued their Final Appraisal Document (FAD) approving the use of midostaurin in first-line treatment of FLT3-mutation-positive acute myeloid leukaemia (AML).

NICE have issued their Final Appraisal Document (FAD) approving the use of midostaurin in first-line treatment of FLT3-mutation-positive acute myeloid leukaemia (AML). Midostaurin, otherwise known as Rydapt®, is produced by Novartis.

Midostaurin is recommended for treating newly diagnosed adults with “acute FLT3-mutation-positive myeloid leukaemia with standard daunorubicin and cytarabine as induction therapy, with high-dose cytarabine as consolidation therapy, and alone after complete response as maintenance therapy”.

Current treatment for AML includes intensive chemotherapy, for those who are fit enough to tolerate it. It involves two phases, induction and consolidation chemotherapy. These aim to induce remission and then ensure undetectable leukaemia cells are destroyed. Midostaurin is to be used in combination with current chemotherapy treatments during the induction and consolidation phases. It is then used alone (monotherapy) for 12 months, taken orally, aiming to prevent the leukaemia from returning. This is known as maintenance therapy.

Midostaurin is a targeted therapy for FLT3-mutation-positive AML. FLT3 mutations are the most common known genetic change in AML, found in around 1 in 4 cases (25% of patients). It is hugely important that patients receive timely cytogenetic testing at diagnosis, so that they can be treated with midostaurin if they are eligible.

Data from the RATIFY clinical trial, involving 717 AML patients with FLT3 mutations, showed significant clinical improvements of treating patients with midostaurin in combination with chemotherapy. Including longer survival, even for patients who eventually relapsed.

NICE acknowledged that there were many benefits of the oral treatment, including improved survival, quality of life and the possibility of inducing remission in first treatment. All of which are welcomed by both patients and clinicians.

It is therefore a very positive outcome for patients that midostaurin has been approved for use in the treatment of FLT3-mutation-positive AML.

Campaigns and Advocacy Director, Zack Pemberton Whiteley, commented:

“Acute Myeloid Leukaemia is a rapidly progressing and often fatal disease. Overall only around 20 percent of patients with AML will survive for five years or more after they are diagnosed. As new treatment options become available, we urgently need to ensure that timely testing is done to match the right patient with the right treatment, especially for those with aggressive forms of the disease.”

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