EU regulators to fast-track Janssen’s multiple myeloma CAR-T

European regulators are to hasten development of Janssen’s CAR-T therapy for patients with multiple myeloma, after promising early-stage trial results.

Janssen, the pharma arm of Johnson & Johnson, said the European Medicines Agency has granted a PRIME (PRIority MEdicines) designation for the investigational B-cell maturation antigen (BCMA) chimeric antigen receptor T-cell (CAR-T) therapy, JNJ-4528.

Janssen has been developing the CAR-T previously known as LCAR-B38M with China’s Legend Biotech following a deal signed in December 2017.

US biotech bluebird bio and partner Celgene are competitors and have two BCMA CAR-T therapies in early stage development for multiple myeloma, while Amgen’s AMG 420 has also emerged as a potential contender with some spectacular remission rates in a small trial announced last September.

But all these drugs are unproven and need more rigorous scientific data to convince regulators to approve them, although bluebird may file data for one of its BCMA CAR-Ts in the next year or so.

GlaxoSmithKline’s GSK2857916 is also considered a competitor. Although it is an antibody it also targets BCMA and has produced some strong data in multiple myeloma, prompting GSK’s chief scientific officer Hal Barron to pledge a rapid filing with regulators late last month.

The first CAR-T therapy accepted by the Chinese regulator for review, Legend has granted Janssen a worldwide licence to jointly develop and market JNJ-4528 in multiple myeloma in a deal where the US biotech paid $350 million up front.

Outside Greater China the companies are splitting costs and profit halfway, while Janssen and Legend have arranged a 30/70% cost-profit sharing agreement within the area.

PRIME offers more interaction and early dialogue to optimise development plans, with the possibility of a faster review, for scientific advances targeting a high unmet medical need.

The EMA made its decision based on results from the phase 1/2 LEGEND-2 study testing LCAR-B38M CAR-T cells, sponsored by Nanjing Legend Biotech.

It also considered results from the phase 1b/2 CARTITUDE-1 study testing JNJ-4528, sponsored by Janssen and conducted in partnership with Legend Biotech USA.

JNJ-4528 is being investigated for patients with multiple myeloma who have received at least three prior treatments, including a proteasome inhibitor, an immunomodulatory drug, and Janssen’s anti-CD38 antibody Darzalex (daratumumab).

Patients must have documented disease progression within 12 months of starting the most recent therapy, or are double refractory to a proteasome inhibitor and immunomodulatory drug.

These patients have few treatment options available and are often faced with poor outcomes.

Sjaak Bot, head of EMEA regulatory affairs at Janssen Biologics, said: “The PRIME designation of this novel BCMA CAR-T therapy highlights the value of regulatory innovation in the European Union.

“We hope to bring this important advance to patients as quickly as possible and this PRIME designation, the first for Janssen, marks an important milestone towards potential market approval.”

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