Zydelig is a new first-in-class drug indicated as a monotherapy for the treatment of adult patients with follicular lymphoma (FL) (a type of non-Hodgkin lymphoma) that are refractory to two prior lines of treatment. 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, critical components of the immune system.
Tony Gavin, Chief Executive at Leukaemia Care stated in a recent news release: “This decision by the SMC is great news for people living with follicular lymphoma in Scotland, and we are thrilled that patients who previously had limited treatment options now have access to idelalisib. We are also greatly encouraged by the SMC’s new approach to reviewing drugs for rare diseases, and are pleased to have been consulted as part of this new ‘ Patient and Clinical Engagement’ process, to be able to provide our, and our patients, perspectives on the acknowledged real-life benefits this novel agent can bring to patients and their families.”
Follicular lymphoma (FL), a B-cell lymphoma, is the most common indolent (slow-growing) form of NHL, accounting for approximately 20 percent to 30 percent of all NHLs. Common signs of disease include enlargement of the lymph nodes in the neck, underarm, stomach, or groin, as well as fatigue, shortness of breath, night sweats, and weight loss. Often, people with FL have no obvious symptoms of the disease at diagnosis.
Approximately 30 percent to 40 percent of patients with FL will eventually develop a transformed lymphoma, which is often more aggressive and usually treated with high-dose chemotherapy along with an autologous stem cell transplant.
Dr. Angus Broom, Consultant Haematologist, Western General Hospital, Edinburgh said in the news release: “It is welcome news that idelalisib is now available for patients in Scotland, as improving quality of life is one of the main goals in the treatment of FL. Access to this treatment offers patients with FL a new option, for those patients unsuitable for chemotherapy.”
SMC’s decision was based on the results from a Phase 2 study (Study 101-09) where the drug agent was evaluated in 125 patients with iNHL who were refractory to rituximab and alkylating-agent-containing chemotherapy. Results from this study showed that in 72 patients, idelalisib reached an overall response rate of 54%, and the average duration of response was not achieved. Adverse side effects included neutropenia, infections, diarrhea/colitis, pneumonitis, increased transaminase (indicator of liver function), rash and pyrexia.
The European Commission granted Marketing Authorisation for idelalisib for the treatment of double refractory FL and chronic lymphocytic leukaemia (CLL) in the European Union, on 19 September 2014.