First STAMP (specifically targeting the abl myristoyl pocket) inhibitor for treatment for chronic myeloid leukaemia (CML), Scemblix®? (asciminib) receives NICE final draft recommendation in Great Britain

• Asciminib final draft recommended by the National Institute for Health and Care Excellence (NICE) for use for treatment of 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
• The final draft recommendation provides access to an innovative treatment option addressing an unmet need for eligible people with CML who commonly develop intolerance or resistance to current treatment with tyrosine kinase inhibitors (TKIs)^1?3
• Asciminib is the first treatment in its class to be authorised in Great Britain

LONDON, June 30, 2022 /PRNewswire/ -- Novartis Pharmaceuticals UK is pleased to announce that eligible patients in England and Wales will soon have routine access to Scemblix^® (asciminib), the first STAMP inhibitor for patients with chronic myeloid leukaemia (CML). The news comes as the National Institute for Health and Care Excellence (NICE) published its Final Appraisal Determination (FAD) recommending asciminib for treatment of 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.¹

"Though outcomes for CML patients have improved, challenges in treatment have continued to have an impact on patients' quality of life, together with the potential to develop resistance or intolerance to their current treatment," commented David FitzGerald, Trustee, CML Support UK. "For this reason, we welcome the addition of asciminib to the treatment pathway, offering a new option for third-line therapy for eligible patients living with CML."

In England and Wales, there are approximately 830 new cases of CML a year, with the majority of patients requiring lifelong treatment and management.^4,5 A switch in treatment is not uncommon for those with CML, with almost half of patients changing the TKI they are on after becoming intolerant or developing resistance.^1,2 Failure rate, however, increases with each line of therapy.²

"Over the past two decades, TKIs have been the mainstay of treatment for CML, however, over time patients often have to switch their treatment due to intolerance or resistance.  A significant number of patients on second-line TKI treatment fail to achieve a satisfactory response after two years and are at risk of disease progression," ⁶ said Dr Dragana Milojkovic, Professor of Practice (Chronic Myeloid Malignancies), Department of Immunology and Inflammation, Imperial College London. "Where the next treatment option has previously been another conventional TKI, today's decision means that clinicians can now consider a novel TKI with an alternative mechanism of action, that may support outcomes for eligible CML patients."

"NICE's positive recommendation for asciminib means that physicians can now consider an innovative treatment for people living with chronic myeloid leukaemia, a long-term debilitating and life-threatening condition," said Heather Moses, Oncology country medical director, Novartis UK. "This milestone strengthens Novartis' legacy in oncology and is evidence of our ongoing commitment to bring forward pioneering, precision treatment options to address unmet needs and help improve quality of life for people living with cancer."

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.

Novartis UK is on Twitter. Sign up to follow @NovartisUK at www.twitter.com/novartisuk

References

1. National Institute For Health And Care Excellence (2022). Final appraisal: https://www.nice.org.uk/guidance/gid-ta10691/documents/final-appraisal-determination-document
2. Akard LP, et al. Clin Adv Hematol Oncol. 2013;11:421?432.
3. Milojkovic D, et al. Br J Haematol. 2021;192:62?74.
4. Cancer Research UK (2021). Chronic Myeloid Leukaemia (C92.1): 2016-2018. Available at: https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/leukaemia-cml/incidence#collapseZero. Accessed: June 2022.
5. Bower H, et al. J Clin Oncol. 2016;34:2851?2857.
6. Cortes J and Lang F. J Hematol Oncol 2021;14:44.