Novartis Receives GB Licence for Scemblix®▼ (Asciminib), the First STAMP Inhibitor for Patients With Chronic Myeloid Leukaemia

Novartis UK has announced that Scemblix^® (asciminib) has been granted Marketing Authorisation in Great Britain by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs), and without a known T315I mutation^.1

Asciminib has been available to specific eligible patients in Great Britain since January 2022, under the MHRA Early Access to Medicines Scheme (EAMS).² It 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 is based on results from the Phase III ASCEMBL trial in patients with Ph+ CML-CP who had experienced resistance or intolerance to at least two TKIs, the ASCEMBL trial^1,3,4 showed that:

• Asciminib nearly doubled the major molecular response (MMR) rate vs. bosutinib at 24 weeks (25% vs. 13% [P=0.029])
• The proportion of patients who discontinued treatment due to adverse reactions was more than three times lower in the asciminib arm (n = 156) vs. patients in the bosutinib arm (n = 76) (7% vs. 25%)
• The most common (incidence ≥ 20%) adverse reactions and laboratory abnormalities in the asciminib arm were, respectively: upper respiratory tract infections and musculoskeletal pain; decrease in platelet and neutrophil counts, decrease in hemoglobin; increase in triglycerides, creatine kinase and alanine aminotransferase (ALT).

“While TKIs 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.”

There are around 830 new cases of CML per year in the UK⁵ and for most patients treatment is lifelong.⁶ Around half of patients treated with a TKI require a change of treatment due to intolerance or developing resistance to treatment.⁷ With each line of treatment, the failure rates increase.^8,9 Asciminib is a first-in-class treatment providing an alternative mechanism of action – an inhibitor specifically targetting the ABL Myristoyl Pocket, known as a STAMP inhibitor.

“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.”