NICE Final Recommendation Of Tasigna (nilotinib) For Some Ph+ Chronic Myeloid Leukaemia Patients Welcomed, UK

The decision by the National Institute for Health and Clinical Excellence (NICE) to recommend Tasigna (nilotinib) in the treatment of imatinib-resistant and imatinib-intolerant Philadelphia Chromosome (Ph+) chronic myeloid leukaemia (CML) was welcomed by Novartis Oncology. Although Dasatinib and high dose imatinib were appraised in the same setting, neither is recommended, with final Guidance expected to be in October 2011.

Panos Alexakos, Oncology General Manager at Novartis UK & Ireland said:

“Today’s Final Appraisal Determination recommending Tasigna is great news for CML patients across England and Wales as it provides access to a treatment option proven to be clinically-effective in this setting. This decision should finally bring clarity on the most clinical and cost-effective second-line treatment for CML patients who are unable to continue with imatinib therapy when it has failed or is not suitable.”

Research revealed, in second line after 24 months of treatment that 87% of patients treated with nilotinib 400mg BD achieved overall survival, 44% achieved complete cytogenic response (CCyR), and over 80% maintained their CCyR.

NICE’s decision on nilotinib reflects their acknowledgement of nilotinib’s clinical and cost-effectiveness and its innovation. Nilotinib, based on imatinib, has been rationally developed to be more specific in its binding to the Bcr-Abl kinase domain, the single known cause of CML.

By being able to target Bcr-Abl more precisely, nilotinib overcomes resistance or intolerance seen with imatinib therapy, resulting in 87% of overall survivals at 24 months. The ability to target Bcr-Abl precisely, results in an improved safety profile compared to TKIs, which have a multi-targeted approach leading to off-target side-effects.

Panos Alexakos said:

“At Novartis Oncology we are committed to ensuring that CML patients in the UK have access to effective life-extending medicines and receive parity of care with other Western European countries. In that regard, we remain hopeful that the Department of Health (DH) and NICE will continue to work closely with the pharmaceutical industry and will focus their efforts on supporting patients and clinicians in the UK in accessing effective treatments in a timely way.”

Novartis Oncology worked closely with the Department of Health (DH) to establish a Patient Access Scheme (PAS) for nilotinib, in order to maximize and ensure patient access to treatment in this setting.

For CML Patients in Wales, who had problems in accessing this second-line therapy for over three years whilst waiting for the outcome of the NICE review process, the news of the decision on nilotinib has been particularly encouraging.

Novartis Oncology therefore hopes that NICE will swiftly carry out its forthcoming review of CML therapies for newly-diagnosed CML patients, keeping patients’ best interest in mind.

CML, one of the four most common types of leukaemia, is the result from an increased production of abnormal (leukaemic) white blood cells by stem cells in the bone marrow. Approximately 560 people are diagnosed with CML in the UK each year; clinical trials indicate, that approximately 16% of CML patients on imatinib discontinue treatment due to unsatisfactory therapeutic outcome, or resistance, and around 6% due to intolerance over an eight year period.

Novartis Oncology first launched imatinib in 2001 for CML.

The development of nilotinib now offers a more effective treatment option available for newly diagnosed CML patients and for those who are resistant to, or intolerant of, imatinib.

Nilotinib was accredited for an award for ‘innovation in research and development in the orphan drug category for rare conditions’ by the judges of the Prix Galien award in 2008.