MHRA approves Novartis treatment for CML  

Stamp of approval

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted Marketing Authorisation to Novartis UK’s treatment for a specific type of cancer. 

The authorisation was granted for Scemblix (asciminib), Novartis UK’s therapy for adults with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) in chronic phase (CP) who have previously been treated with two or more tyrosine kinase inhibitors (TKIs), and without a known T315I mutation. 

Certain eligible patients have already been able to access asciminib in Great Britain under the MHRA’s Early Access to Medicines Scheme (EAMS). The therapy is currently under review by the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC), with guidance anticipated in the coming months. 

The authorisation of asciminib was based on results of a Phase III trial including 233 patients with Ph+ CML-CP who had experienced resistance or intolerance to at least two TKIs1. The trial showed that at 24 weeks patients on asciminib has nearly double the major molecular response (MMR) versus the small molecule drug bosutinib. More so, the number of patients who discontinued treatment due to adverse reactions was more than three times lower in the asciminib arm compared to patients in the bosutinib arm (7% vs. 25%). Adverse reactions included upper respiratory tract infections and musculoskeletal pain; decrease in platelet and neutrophil counts, decrease in haemoglobin; increase in triglycerides, creatine kinase and alanine aminotransferase (ALT).   


According to Cancer Research UK there are around 830 new cases of CML every year in the UK2. More so, it’s common for patients to have to change treatment from a TI due to intolerance or developing resistance to treatment.  

Official comments  

While TKIs have revolutionised treatment for CML since their introduction, thousands of people living with CML in the UK need additional treatment options. Over time, people living with CML can develop intolerance or resistance to their current treatment, and until now the alternative has been a treatment with a similar mechanism of action,” said Professor Jane Apperley, Chair of the Department of Haematology and the Chief of Service for Clinical Haematology at the Imperial College of London. “With its innovative mechanism of action, asciminib is an important and welcome addition to the treatment options available for third-line therapy, that may support outcomes for eligible patients living with CML.” 

“Today’s announcement builds on Novartis’ legacy and commitment to people living with cancer in the UK, bringing forward an innovative treatment option to address high unmet needs for eligible patients living with chronic myeloid leukaemia, a long-term debilitating and life-threatening condition” said Heather Moses, Oncology country medical director, Novartis UK. “We are proud that Great Britain is one of the first countries in Europe where regulatory authorisation means that physicians can now consider asciminib where previously, adequate treatment options were limited.” 




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