CAR-T therapies leap towards EU approval

The stage has been set for approval of the first chimeric antigen T-cell therapies (CAR-T) in Europe, after Novartis’ Kymriah and Gilead’s Yescarta both won backing from the Committee for Medicinal Products for Human Use (CHMP).

The Committee is recommending approval of Kymriah for treatment of B cell acute lymphoblastic leukemia (ALL) and diffuse large B cell lymphoma (DLBCL), and Yescarta for DLBCL and primary mediastinal B cell lymphoma (PMBCL).

CAR-T therapy offers a new treatment approach in that it is specifically manufactured for each individual patient. During the process, T cells are drawn from a patient's blood and reprogrammed in the lab to create T cells that are genetically coded to hunt the patient's cancer cells.

Earlier this year, NHS England chief executive Simon Stevens indicated that the NHS was preparing for the introduction of CAR-T this year, offering eligible patients a ‘ground-breaking’ approach to treating cancer. But he also stressed manufacturers "need to set fair and affordable prices so treatments can be made available to all who need them."

Yescarta and Kymriah are currently being assessed by the National Institute for Health and Care Excellence (NICE) for use on the NHS in England. A decision on both drugs is expected by late 2018.

"The development of CAR-T therapy is one of the most promising advances in blood cancer treatment in decades, and while it would initially only be offered to patients who have run out of other options, it offers the genuine chance of a cure for these patients," noted Dr Alasdair Rankin, director of research at the blood cancer research charity Bloodwise.

The decision "means that one of the most exciting types of blood cancer treatment we’ve ever seen has taken another step towards becoming available on the NHS. The next step is for NICE to decide whether to make these treatments available and then for the NHS to get the complex infrastructure in place that CAR-T relies on."

"The complexity involved in the delivery of CAR T-cell therapy is going to require all of us – patients, doctors, the NHS and industry – to think differently," said Mari Scheiffele, general manager of Novartis Oncology UK & Ireland. "We continue to work together with the NHS to prepare and enable them to adopt this new therapy for UK patients as soon as possible after license. We are proud to be playing our part to deliver this innovative therapy to patients."

The CHMP has also recommended an extension to the therapeutic indication for Roche’s RoActemra (tocilizumab) to include the treatment of CAR-T-cell-induced cytokine release syndrome (CRS), a known serious side effect of CAR-T cell therapies.

Elsewhere, six other therapies were backed for European approval: Ablynx’ Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura; Ultragenyx’ Mepsevii (vestronidase alfa) for mucopolysaccharidosis type VII; Baxalta’s Veyvondi (vonicog alfa) for von Willebrand disease; Jazz Pharmaceuticals’ Vyxeos (daunorubicin/cytarabine) for acute myeloid leukaemia; Grunenthal’s Duzallo (lesinurad/allopurinol) for hyperuricaemia in patients with gout; and Ulipristal Acetate Gedeon Richter for the pre-operative treatment of uterine fibroids.