Gilead's CAR-T cell therapy Yescarta gains EU clearance; meets first resistance from NICE

Gilead Sciences' Kite unit announced that the European Commission approved Yescarta (axicabtagene ciloleucel) as a treatment for adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy. The company noted that the CAR-T cell therapy is first to receive clearance for two types of aggressive non-Hodgkin lymphoma (NHL).

The approval came at the same time as the European Commission approved Novartis' CAR-T cell therapy Kymriah (tisagenlecleucel) for blood cancer indications, including relapsed or refractory DLBCL after two or more lines of systemic therapy. Kymriah also gained authorisation to treat paediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukaemia that is refractory, in relapse post-transplant or in second or later relapse.

The approval of Yescarta was supported by data from the ZUMA-1 trial in adults with refractory aggressive NHL. In the study, 72 percent of patients who received a single infusion of Yescarta responded to therapy, with 51 percent achieving a complete response at a median follow-up of 15.1 months. Gilead at one year following infusion, 60 percent of patients were alive and the median overall survival had not been reached.

Yescarta was approved in the US last year for certain patients with large B-cell lymphoma, with Gilead pricing the therapy at $373 000. For related analysis, see ViewPoints: Gilead preps for its European CAR-T adventure.

On Tuesday, in a setback for Gilead, the National Institute for Health and Care Excellence released draft guidance recommending against routine NHS funding of Yescarta in its two approved indications. The agency noted that while the therapy has demonstrated good response rates, overall survival and progression-free survival, there are no direct data comparing it to salvage chemotherapy. NICE said that as a result, the exact size of the benefit of Yescarta compared with salvage chemotherapy is unknown.

According to NICE, although Yescarta meets its criteria to be considered a life-extending treatment at the end of life, all of its cost-effectiveness estimates are above the range normally considered to be a cost-effective use of NHS resources. The agency noted that the price of the product "was submitted as commercial in confidence," adding that Gilead had also proposed a "commercial arrangement," but disclosed no further details. NICE added that the product does not meet the criteria for inclusion in the Cancer Drugs Fund.

Gilead said it was "in ongoing discussions with NICE to identify appropriate treatment comparators which can clarify how cell therapy may be made available to patients in the UK." Yescarta is the first CAR-T therapy to be assessed by NICE, although the agency is currently reviewing Kymriah.

Commenting on the news, Raj Chopra, head of cancer therapeutics at The Institute of Cancer Research, said "if we're going to see CAR T therapy widely available on the NHS, we need to find ways to reduce the costs." Chopra added "I hope NICE and the manufacturer will be able to work together both to bring the costs of treatment down and continue to build a stronger evidence base for it, so it can be made available to NHS patients as quickly as possible."

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