Pharmaceutical company halts access to CAR-T cell treatment for UK myeloma patients

Pharmaceutical company Janssen has made the decision not to progress its CAR-T cell treatment, cilta -cel (ciltacabtagene autoleucel), (Carvykti™) for UK myeloma patients at this time.

The novel treatment was in the process of being assessed by the National Institute of Health and Care Excellence (NICE), to make the treatment available on the NHS.

But, for the time being, the pharmaceutical company Janssen has opted not to pursue approval from NICE.

Manufacturing CAR-T product is very complex, and it is believed that it is not going to be possible to produce the supply needed to support a programme in the UK.

Janssen’s decision not to progress with the NICE appraisal for approval of the treatment on the NHS will not affect clinical trials.

Dr Sophie Castell, Chief Executive at Myeloma UK, said:

“We know this is devastating news for patients, families, friends and the medical professionals who have been working so hard to prepare for the launch of CAR-T cell treatments in myeloma.

“We are working with the pharmaceutical company Janssen to find out more about the reasons behind this decision. While this is undeniably a major blow, we want the myeloma community to know that we will do all we can to deliver CAR-T cell treatments for patients in the UK.

“While today’s news will be upsetting, there are reasons for hope. In the years since CAR-T cell treatments was first trialled, huge strides have been made in developing other life-extending treatments with new mechanisms of action. We will keep striving to ensure these promising treatments and the many more in the pipeline for multiply relapsed patients – including elranatamab, which is due to be appraised by NICE this year – are made available to those who need them most. Giving patients access to the latest, most effective treatments remains our top priority.”

Prof Graham Jackson, Chief Clinical and Scientific Advisor at Myeloma UK, added:

“The entire medical community is deeply disappointed by this news, but we will continue to do all we can to bring novel treatments, clinical trials and new innovations to the UK. And, of course, we will continue to work with all stakeholders to get CAR-T cell treatments for myeloma to the UK as soon as possible.”

CAR-T cell treatment uses the body’s own immune system to kill myeloma cells. CAR-T cells are made by taking T-cells (a type of immune cell) from the patient’s blood and genetically modifying them in a laboratory, so they are much better at finding and killing myeloma cells. The new CAR-T cells are then given back to the patient. The CAR-T cells can then find and kill the myeloma cells.

Although the CAR-T cell treatments for myeloma are not curative they have the potential to deliver longer remission times and are designed to be a one-off treatment giving patients time off treatment.

Results of trials with anti-myeloma CAR-T cell treatments have so far been impressive, delivering good response rates and remission times in heavily pre-treated patients (on average six lines of treatment) with treatment-resistant myeloma.

It is hoped that the results will be even more impressive when given as the first or second line of treatment. Trials in these groups of patients are ongoing.

Why was the submission withdrawn?

The pharmaceutical company that manufactures cilta-cel didn’t withdraw the submission due to efficacy or safety concerns.

CAR-T cell treatments are very complex and made in a different way from other myeloma treatments. Different materials, processes and controls are needed to make and deliver these drugs. Issues with the supply of CAR-T cell treatments have increased as demand has grown globally.

The manufacturing issues have impacted the growth of all CAR-T cell treatments, not just cilta-cel.

What does this decision mean for myeloma patients in the UK?

The decision means that cilta-cel will not launch in the UK this year.

The National Institute of Health and Care Excellence was evaluating whether cilta-cel should be made available on the NHS to treat myeloma patients who had received three or more previous lines of treatment. Therefore, NICE could have approved the treatment later this year.

Although cilta-cel will not be available through the NHS, patients in the UK may have the opportunity to access cilta-cel or other novel immunotherapies via clinical trials. Multiply relapsed patients may also be able to access novel treatments like belantamab mafodotin (Blenrep®) and elranatamab through compassionate access schemes.

Will CAR-T cell treatments come to the UK?

The pharmaceutical company manufacturing cilta-cel is still developing the drug. Clinical trials are ongoing and the pharmaceutical company could seek NHS approval for cilta-cel later.

There are also other CAR-T cell treatments in development, including ide-cel which is approved for use in the US. The manufacturer of ide-cel, Bristol Myers Squibb, halted its UK drug approval plans in 2020, due to a combination of factors including the impact of the pandemic on its clinical trial delivery.

CAR-T cell treatments are not the only innovative myeloma treatments in development. This year NICE will evaluate novel T-cell engagers (e.g. elranatamab) and antibody-drug conjugates (e.g. belantamab mafodotin) for myeloma.

What will happen next?

Myeloma UK will continue to work with clinicians, pharmaceutical companies, NICE and the NHS to advocate for myeloma patients and ensure they have access to treatments.

We will be writing to the pharmaceutical company and meeting with clinicians to understand what can be done to speed up UK access to CAR-T cell treatments.