Cilta-cel is an investigational BCMA-directed CAR-T therapy in development for the treatment of adults with relapsed and/or refractory multiple myeloma, a long time focus for Janssen.
The design comprises a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies. BCMA is a protein that is highly expressed on myeloma cells.
Accelerated assessment is considered when a medicinal product is expected to be of major public health interest and therapeutic innovation and can significantly reduce the review timelines to evaluate an MAA.
The cilta-cel MAA, which is targeted for submission in the first half of 2021, is supported by the positive results from the Phase 1b/2 CARTITUDE-1 study recently presented at the American Society of Hematology (ASH) 2020 Annual Meeting, said Janssen.
This accelerated approval milestone in Europe follows the December 2020 announcement by the company of the initiation of the rolling submission of its Biologics License Application (BLA) for cilta-cel to the US Food and Drug Administration (FDA).
In December 2017, Janssen entered into an exclusive worldwide license and collaboration agreement with Legend Biotech to develop and commercialize cilta-cel.
In May 2018, Janssen initiated a Phase 1b/2 CARTITUDE-1 trial (NCT03548207) to evaluate the efficacy and safety of cilta-cel in adults with relapsed and/or refractory multiple myeloma, informed by the LEGEND-2 study results.
In April 2019, cilta-cel was granted PRIME (PRIority MEdicines) designation by the EMA.
PRIME offers enhanced interaction and early dialogue with developers of promising medicines, to optimize drug development plans and speed up evaluation of cutting-edge, scientific advances that target a high unmet medical need.
In February 2020, the EU Commission granted orphan designation for cilta-cel.