Leukaemia patients granted early access to investigational treatment 

Novartis UK has announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has given a positive scientific opinion for the investigational treatment asciminib to be made available to appropriate patients under the UK Early Access to Medicines Scheme (EAMS).1 

What does this mean? 

The decision means that adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML- CP) without T315I mutation who have previously been treated with two or more tyrosine kinase inhibitors (TKIs) may now potentially gain access to pill while the relevant regulatory bodies continue to review the Marketing Authorisation Application. 


In the UK, there are around 830 new cases of CML per year2 and for a majority of patients treatment is lifelong, with frequent switches of TKI treatment being common. Approximately half of patients require a change of treatment due to intolerance or developing resistance to treatment, and with each line of treatment the failure rates increase.3-5 

The MHRA Early Access to Medicines Scheme (EAMS) aims to give patients with clear unmet medical need due to a life-threatening condition such as CML access to innovative medicines before any decision on their formal marketing authorisation.6 

Official comments 

“Despite several TKIs being available in the UK, thousands of people living with CML still need additional treatment options due to compromising side-effects or lack of efficacy of their current treatment,” said Dr Dragana Milojkovic, Professor of Practice (Chronic Myeloid Malignancies) in the Department of Immunology and Inflammation at Imperial College London. “People living with CML can develop treatment resistance and intolerance over time, requiring a switch to an alternative treatment, usually another TKI. The introduction of asciminib, which has an innovative mechanism of action is a very welcome and necessary addition to the treatment options available for third line therapy.” 

“This decision allows eligible patients in the UK early access to our investigational treatment, and enables additional real-world evidence to be collected on its efficacy/safety profile while the MHRA completes its full Marketing Autorisation Application review,” said Roland Kreissig, recently-appointed Oncology General Manager, Novartis UK and Ireland. “Novartis has once again demonstrated its long-standing commitment to bold science with the aim of addressing the unmnet needs of people living with chronic myeloid leukaemia.” 


  1. UK Medicines and Healthcare product Regulatory Agency (MHRA), EAMS scientific opinion for asciminib, available via https://www.gov.uk/government/publications/asciminib-in-the-treatment-of-chronic-myeloid-leukaemia.
  2. Cancer Research UK, Chronic myeloid leukaemia(CML) statistics (2016-2018 average for UK), ,https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer- type/leukaemia-cml. 
  3. MilojkovicD, et al. Br J Haematol, 2021;192:62–74. 
  4. Akard LP, et al. Clin Adv HematolOncol. 2013;11:421–432.
  5. Jabbour E, et al. Clin Lymphoma Myeloma Leuk2015;15:323–334.
  6. UK Medicines and Healthcare product Regulatory Agency (MHRA), Guidance: Early Access to Medicines Scheme (EAMS), available via https://www.gov.uk/guidance/apply-for-the-early-access- to-medicines-scheme-eams#overview. 

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