MSAC rejects funding for Janssen’s CAR-T therapy despite magnitude of results from clinical trial

Pharma News: Janssen’s ciltacabtagene autoleucel (cilta-cel) is a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T (CAR-T) cell therapy for treatment of patients with multiple myeloma (MM) who are refractory or have failed more than three lines of prior therapy.

The Medical Services Advisory Committee (MSAC) has not supported the public funding of Carvykti (cilta-cel) through the National Health Reform Agreement despite the magnitude of the clinical trial results and support from Myeloma Australia.

MSAC said it “did not accept that cilta-cel is comparatively safe, effective and cost-effective over the modelled time horizon. MSAC also considered the low level of clinical evidence in support of cilta-cel to be unacceptable in the context of late-line treatment [at least three prior lines of therapy, including a PI (bortezomib, carfilzomib); an IMiD (lenalidomide, pomalidomide), and an anti-CD38 antibody (daratumumab)] where other treatment options are available, and the prevalence of relapsed or refractory multiple myeloma (RRMM) being clearly beyond that of a rare disease, with a large and uncertain financial impact.”

Patients in CARTITUDE-1 were relatively healthier and able to tolerate intensive treatment, according to MSAC. This may not reflect the eligible population in Australia and may bias the effect in favour of cilta-cel.

MSAC also noted the recent Pharmaceutical Benefits Advisory Committee (PBAC) recommendations for new triple therapies for patients with RRMM “which could result in cilta-cel moving further down the line of therapies… where patients are less well when they receive cilta-cel therapy which may mean reduced effectiveness of cilta-cel therapy for the same cost and toxicities, representing less value for money.”

Janssen Australia said in a statement that the company is disappointed in MSAC’s decision. “Janssen acknowledges that CARTITUDE 1 is a single arm study, however the magnitude of benefit seen in the trial is substantial and clinically meaningful, and the unmet need in this patient population is very high, such that the evidence presented is appropriate to support a funding recommendation.

“The latest data from CARTITUDE 1 shows that more than two years after treatment, 55% of patients have no cancer progression and 70% remain alive. This is unheard of for patients receiving their fourth line of therapy, where median survival is just 5.4 months. Janssen also notes that the advice of MSAC conflicts with their approval of previous CAR T-cell therapies which were based on single-arm clinical trials.”

“In clinical trials, ciltacabtagene autoleucel delivered survival rates not previously seen in heavily pre-treated myeloma patients. We remain committed to working with all stakeholders to ensure equitable and timely access to CAR T-cell therapy in Australia.”

In fact, in an analysis of the data from the 2-year follow-up of the pivotal CARTITUDE-1 trial, presented at the 2022 ASCO Annual Meeting, the investigators concluded “At approximately 28 months median follow-up, patients treated with ciltacabtagene autoleucel maintained deep and durable responses, observed in both standard and high-risk subgroups. The risk/benefit profile of ciltacabtagene autoleucel remained favourable with longer follow-up.”

MSAC has previously supported public funding of three other CAR-T therapies since 2019 for lymphoma and/or leukaemia: Gilead’s Yescarta and Tecartus, and Novartis’ Kymriah.

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