People with chronic myeloid leukaemia granted early access to Novartis’ investigational treatment

People with chronic myeloid leukaemia granted early access to Novartis’ investigational treatment

• The Medicine and Healthcare products Regulatory Agency (MHRA) grants positive scientific opinion on use of asciminib as 3rd-line treatment option for adults with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML-CP) without T315I mutation under the UK Early Access to Medicines Scheme (EAMS)

• With its novel mechanism of action, asciminib is the first STAMP inhibitor available to UK patients

London, January 24, 2021 - 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 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 asciminib while the relevant regulatory bodies continue to review the Marketing Authorisation Application.

“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), Department of Immunology and Inflammation, 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.”

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

“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.”