The National Institute for Health and Clinical Excellence (NICE) has published final guidance recommending nilotinib for the first-line treatment of chronic phase Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML).

 This publication completes the appraisal process for nilotinib, allowing physicians to prescribe this cost-effective treatment option with superior efficacy vs. the standard of care, imatinib.

It also means that Ph+ CML patients are now entitled to access a clinically effective and generally well tolerated treatment at the best possible time to significantly improve outcomes.

Following the NICE recommendation for nilotinib in imatinib-resistant or intolerant (second-line) patients in January 20124, this guidance in newly diagnosed patients makes nilotinib the only treatment to be recommended for both first- and second-line Ph+ CML. Imatinib and dasatinib where also assessed in this latest NICE appraisal but only nilotinib and imatinib were recommended for first-line treatment of CML.

Sandy Craine, Co-Founder of the CML Support Group commented: “The publication of this final guidance will give hundreds of people in the UK diagnosed with CML greater assurance that they can survive this rare and life-threatening disease with the chance to live out a normal life as active members of society. Patients will now have equality of access to nilotinib alongside patients in Scotland and other European member states. This will increase their chances of successful treatment and is a significant milestone.”

The Scottish Medicines Consortium (SMC) recommended nilotinib for the treatment of newly diagnosed Ph+ CML patients in 2011, and this NICE guidance will now expand the availability of the drug to patients in England, Wales and Northern Ireland.

In order to ensure that Ph+ CML patients are able to access nilotinib, Novartis Oncology worked closely with the Department of Health (DH) to establish a Patient Access Scheme (PAS) for the drug.

“Publication of the final guidance for nilotinib demonstrates what can be achieved through successful collaboration between the pharmaceutical industry and regulatory bodies,” said Mr Panos Alexakos, Oncology General Manager, Novartis UK & Ireland. “By putting patients’ healthcare needs at the core of our values, a significant step has been taken to ensure that the right treatments can be offered safely to patients and at the right time.”

Nilotinib has been designed to be more specific in its binding to the Bcr-Abl kinase domain, of which a genetic mutation of this protein is central to the cause of CML6,7. As a result, nilotinib reduces the rate of progression to more advanced stages of the disease and is proven to generate earlier and higher major molecular response (MMR) rates than the current standard of care, imatinib1,2. Follow-up study results show CCyR, MMR and CMR rates for nilotinib in this patient group continue to be superior to imatinib at 24 months2.

Nilotinib received final guidance by NICE in January 2012 for use in the treatment of Ph+ CML patients who have become intolerant or resistant to imatinib4.

Novartis Oncology has a long-standing commitment to advancing treatment in CML. The Novartis CML therapy imatinib revolutionised treatment of the disease when it was first launched in 20018. Now, with the development of nilotinib, a more targeted and effective treatment option is available for newly diagnosed patients and for those who are resistant to or intolerant of imatinib.

Source: Novartis