An accelerated review will be given an application for European Union approval of ciltacabtagene autoleucel (cilta-cel), a potential CAR T-cell therapy for heavily pre-treated multiple myeloma patients, its developer, Janssen, announced.
An accelerated assessment, given to medications expected to have a major impact on public health, shortens the regulatory review process in Europe to 150 days from the standard 210 days. The decision to grant such a review is made by the Committee for Medicinal Products for Human Use (CHMP), a branch of the European Medicines Agency (EMA).
Janssen expects to file its approval request — in the form of a marketing authorization application — by the close of June. In the U.S., the company recently initiated a rolling submission requesting cilta-cel’s approval for this same patient group.
“We are excited that the potential clinical benefit of cilta-cel is being recognised and now look forward to working with the EMA to bring this highly innovative treatment to patients in need,” Saskia De Haes, vice president of EMEA Regulatory Affairs at Janssen, said in a press release.
Cilta-cel is an experimental CAR T-cell therapy — a form of immunotherapy that uses a patient’s own immune T-cells, modified in the lab to better recognize and eliminate cancer cells. It is being jointly developed by Janssen and Legend Biotech for adults with relapsed or refractory multiple myeloma.
Modified T-cells in cilta-cel are specifically engineered to target and kill myeloma cells containing the B-cell maturation antigen (BCMA). This protein is found at high levels on the surface of myeloma cells.
Janssen’s application is supported by data from the ongoing, open-label CARTITUDE-1 Phase 1b/2 trial (NCT03548207), which is assessing the safety and efficacy of cilta-cel in adults with relapsed or refractory multiple myeloma.
Those enrolled have received at least three prior lines of therapy — including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and an anti-CD38 antibody — or failed to respond to treatment with a PI and an IMiD.
The latest data from CARTITUDE-1, presented at a December hematology meeting, included one-year findings in 97 patients. A single infusion of cilta-cel was seen to reduce tumor burden in nearly all (97%) these people, and more than half (67%) attained a deep complete response (no detectable cancer).
Treatment responses were first evident after a median of one month, and tended to improve over time, with 72% of study participants having ongoing responses after one year.
Cilta-cel’s safety profile in CARTITUDE-1 has been consistent with that of previous studies, with the most common being low levels of blood cells that include neutrophils, platelets, red blood cells, leukocytes, and lymphocytes. At the time of these analyses, a total of 14 patients had died, including six from treatment-related side effects.
“Multiple myeloma is a rare, incurable cancer, and has long been an area of focus for Janssen. We are deeply committed to improving outcomes for patients with multiple myeloma, with a goal of delivering innovations that have the potential to expand current remission periods and improve quality of life,” said Sen Zhuang, MD, PhD, vice president of Clinical Research Development at Janssen.
The EMA previously granted PRIME (PRIority MEdicines) and orphan drug designations to cilta-cel, both meant to accelerate its development by providing regulatory support, financial benefits, and a marketing exclusivity period of 10 years upon regulatory approval.