Scottish Medicines Consortium (SMC) recommend the use of midostaurin for adult acute myeloid leukaemia patients

Scotland receives a positive recommendation by the SMC for the use of FLT3-mutation-positive targeted therapy midostaurin in first-line AML treatment.

The Scottish Medicines Consortium (SMC) have today recommended the use of midostaurin in first-line treatment for adult acute myeloid leukaemia (AML) patients, who are FMS-like tyrosine kinase 3 (FLT3) mutation-positive.

Midostaurin, brand name Rydapt, is produced by pharmaceutical company Novartis.

The treatment is recommended for use “in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy, and for patients in complete response followed by midostaurin single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FMS-like tyrosine kinase 3 (FLT3) mutation-positive.”

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 taken orally and 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, aiming to prevent the leukaemia from returning. This is known as maintenance therapy.

In stage III trials, patients treated with midostaurin in combination with chemotherapy showed significant improvements in survival compared to chemotherapy alone.

The SMC decision is, therefore, very positive for FLT3-mutation-positive patients. 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.

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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