Gilead Sciences Inc. announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion on the company’s Marketing Authorization Application (MAA) for Zydelig (idelalisib 150 mg film-coated tablets), a first-in-class treatment for patients with chronic lymphocytic leukemia (CLL) and follicular lymphoma (FL), the most common subtype of indolent non-Hodgkin lymphoma (iNHL). The CHMP opinion supports the use of Zydelig 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 in CLL patients in the presence of 17p deletion or TP53 mutation in patients unsuitable for chemo-immunotherapy and also as monotherapy for the treatment of adult patients with FL that is refractory to two prior lines of treatment. The CHMP’s recommendation will now be reviewed by the European Commission, which has the authority to approve medicines for use in the 28 countries of the European Union (EU).
CLL and FL are slow-growing incurable blood cancers that can lead to life-threatening complications such as anemia, serious infection and bone marrow failure requiring treatment. Relapse commonly occurs after initial chemo-immunotherapy and many patients with relapsed CLL or FL are unable to tolerate chemotherapy, which may limit their treatment options.
The CHMP positive opinion for Zydelig is based on data from two clinical trials – Study 116 and Study 101-09. Study 116, a pivotal Phase 3 trial, investigated the efficacy and safety of Zydelig in combination with rituximab in patients with previously treated CLL. The Phase 2 101-09 study assessed the efficacy and safety of Zydelig in patients with iNHL who are refractory to rituximab and alkylating agents. Results of Study 116 and Study 101-09 were published in The New England Journal of Medicine in March 2014.
The most commonly reported adverse reactions (incidence =20%) were diarrhea, pyrexia, fatigue, nausea, cough, abdominal pain and chills. Additionally, grade 3 or 4 elevations in ALT and AST (indicators of liver function) have been observed in clinical trials of Zydelig. Grade 3 or 4 pneumonitis and Grade 3 or 4 diarrhea/colitis were also observed in some patients in the clinical trials.
Zydelig is an investigational product and its safety and efficacy have not been established in the European Union.
Date: July 25, 2014