The Medicines and Healthcare products Regulatory Agency (MHRA) grants marketing authorisation for use of asciminib for adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors, and without a known T315I mutation
Asciminib is the first treatment in its class to be authorised in Great Britain
London, June 16, 2022 - 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 trial1,3,4 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.”
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About Novartis Commitment to CML Novartis has a long-standing scientific commitment to patients living with CML. For more than 20 years, our bold science has helped transform CML into a chronic disease for many patients. Despite these advancements, we’re not standing still. We continue to research ways to target the disease, seeking to address the challenges with treatment resistance and/or intolerance that many patients face. Novartis also continues to reimagine CML care through its commitment to sustainable access for patients and collaboration with the global CML community.
About Novartis Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we strive to use innovative science and digital technologies to create treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach more than 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world.
In the UK, we employ approximately 1,500 people to serve healthcare needs across the whole of the UK, as well as supporting the global operations of Novartis. Since 2014, Novartis has invested over £200 million in R&D and is a leading sponsor of clinical trials, in the UK. For more information, please visit www.novartis.co.uk.
Notes to Editors: ▼ This medicine is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse events. Reporting forms and information can be found at mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store.
Novartis, Data on file, Notification of MHRA Marketing Authorisation, June 2022.
Rea D, et al. Blood. 2021. DOI: 10.1182/blood.2020009984. PMID: 34407542.
Mauro, JM, et al. Efficacy and Safety Results from ASCEMBL, a Multicenter, Open-Label, Phase 3 Study of Asciminib, a First-in-Class STAMP Inhibitor, Vs Bosutinib in Patients with Chronic Myeloid Leukemia in Chronic Phase after ≥2 Prior Tyrosine Kinase Inhibitors: Update after 48 Weeks. Oral presentation 310 at: American Society of Hematology (ASH) Annual Meeting; Dec. 11, 2021.