RA dayNICE has recommended idelalisib (Zydelig) as a treatment option in combination with rituximab for adults with chronic lymphocytic leukaemia (CLL) who have relapsed early (<24 months). This combination has also been recommended for untreated CLL patients with markers for aggressive disease (genetic abnormalities called 17p deletion and/or TP53 mutation). As assessed by the NICE Committee, this decision indicates that idelalisib plus rituximab can offer significant health-related benefits.

CLL is the most common form of adult leukaemia in the UK, affecting approximately 28,140 people. The disease can lead to life-threatening complications, such as anaemia, serious infection and bone marrow failure, and has a significant negative impact on patients’ quality of life.

“This is great news for patients with CLL,” said Chris West, Spokesperson for Bloodwise, the UK’s biggest blood cancer charity. “Coinciding with National Blood Cancer Awareness Month, NICE’s recommendation of idelalisib is a very welcome development for adult patients with CLL in England and Wales, particularly when there is such uncertainty around the future availability of many blood cancer treatments.”

Chemo-immunotherapy is usually the first choice for treating CLL after ‘watch and wait’, however most patients eventually relapse and need further treatment. For patients with aggressive disease that relapses within 2 years of chemo-immunotherapy treatment options are very limited. Also 5% of patients at diagnosis and up to 10% at treatment initiation have specific genetic abnormalities, and for these patients most conventional chemo-immunotherapy treatments are not very effective and deliver poor responses with relatively short duration. 

"Idelalisib provides a much needed option for those patients with CLL in whom conventional chemotherapy-based treatment are not very effective, including when the disease relapses quickly after previous therapy and for previously untreated patients with a molecular switch (17p deletion or TP53 mutation) that prevents chemotherapy being effective," said Professor Peter Hillmen, Consultant Haematologist, NHS Leeds Teaching Hospitals. "Previously these patients have been faced with very limited, ineffective treatment, and had a much shorter survival than other patients with CLL. However NICE's decision means that these patients will soon be able to access through the NHS an effective, chemotherapy-free treatment option that directly targets the leukaemia leading to improved survival."

Idelalisib, which received Marketing Authorisation from the European Commission one year ago, has been available to a restricted patient population in England through the Cancer Drugs Fund since January. This decision by NICE signals idelalisib’s long-term availability for eligible patients, and brings England and Wales in line with the NHS in Scotland, as the Scottish Medicines Consortium (SMC) approved idelalisib for use for Scottish NHS patients in March 2015. 

Idelalisib is an oral inhibitor of phosphoinositide 3-kinase (PI3K) delta, a protein that plays a role in the activation, proliferation and viability of B cells, a critical component of the immune system. PI3K delta signalling is active in many B-cell leukaemias and lymphomas, and by inhibiting the protein, idelalisib blocks several cellular signalling pathways that drive B-cell viability. Idelalisib is indicated in combination with rituximab for the treatment of adult patients with CLL who have received at least one prior therapy, or as first-line treatment for CLL patients in the presence of 17p deletion or TP53 mutation in patients unsuitable for chemo-immunotherapy. Idelalisib is administered orally twice-daily and is available as 150mg and 100mg dose strengths. The European Commission granted Marketing Authorisation for idelalisib for the treatment of two incurable blood cancers, double refractory FL and chronic lymphocytic leukaemia (CLL) in the European Union, on 19 September 2014.