Rare blood cancer treatments recommended for approval by the EMA
The European Medicines Agency has approved two new medicines, ibrutinib and idelalisib, for adult patients with rare blood cancers.
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Two new medicines for adult patients with rare blood cancers have been given initial authorisation by the European Medicines Agency (EMA).
The EMA recommended ibrutinib (Janssen-Cilag’s Imbruvica) and idelalisib (Gilead Science’s Zydelig) for approval after its scientific committee, the Committee for Medicinal Products for Human Use (CHMP), issued positive opinions for both medicines on 24 July 2014.
The medicines now need approval from the European Commission, a process which takes around two months after the EMA’s initial authorisation. The product literature, or summaries of product characteristics, will be published after the marketing authorisation has been granted.
The EMA believes the two drugs have potential as new treatment options, especially in cases where previous treatments have stopped working as they act in novel ways compared with other medicines.
Ibrutinib, an orphan medicine administered in the form of 140mg hard capsules, inhibits Bruton’s tyrosine kinase, a signalling molecule of the B-cell antigen receptor (BCR) and cytokine receptor pathways.
It is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL). It is also indicated for adults with chronic lymphocytic leukaemia (CLL) who have received at least one prior therapy, or as first-line treatment in patients with a specific genetic mutation — the presence of 17p deletion or TP53 mutation — which makes them unsuitable for chemo-immunotherapy.
The EMA says ibrutinib improves progression-free survival in CLL, as shown in a randomised controlled trial against ofatumumab. The benefits in MCL are improved overall response rate and duration of response, as shown in a single-arm study.
The most common side effects for ibrutinib include pneumonia, sinusitis, neutropenia, thrombocytopenia, anaemia, headache, haemorrhage, bruising, diarrhoea, vomiting, stomatitis, pyrexia and peripheral oedema.
The second drug idelalisib, administered in the form of 100mg and 150mg film coated tablets, inhibits phosphatidylinositol 3 kinase p110δ (PI3Kδ), which is hyperactive in B cell cancers and is central to multiple signalling pathways that drive proliferation of malignant cells in lymphoid tissues and bone marrow.
It 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 in the presence of 17p deletion or TP53 mutation in patients unsuitable for chemo-immunotherapy. It is also indicated as monotherapy for adult patients with follicular lymphoma (FL) that is refractory to two prior lines of treatment.
In CLL, idelalisib in combination with rituximab improves progression-free survival, as shown in a randomised controlled trial against placebo. The benefits in FL of idelalisib monotherapy are in terms of overall response rate and duration of response, as shown in a single-arm study. The most common side effects are infections, neutropenia, increased transaminase, increased triglycerides, diarrhoea/colitis, rash and pyrexia.
According to the EMA, CLL affects around 3 in 10,000 people and FL 3.7 in 10,000 people in the EU, equating to fewer than 200,000 people for each disease. CLL is the most common form of adult leukaemia in the EU and remains an incurable disease.
“Treatments available generally induce remission, however, the disease returns in nearly all patients,” the EMA says. “For follicular lymphoma, a need remains for new medicines for patients who have failed prior treatment.”
Mantle cell lymphoma is a very rare cancer, with some 15,000 people affected in the EU. “Current available treatments for mantle cell lymphoma do not cure the disease and there is no standard-of-care treatment for the cancer when it comes back or is not responding after previous treatments,” the EMA says.
Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2014.20066021
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