J&J halts Carvykti UK launch but is manufacturing to blame?

Patient advocacy group Myeloma UK believes supply issues have caused Johnson & Johnson to not progress its CAR-T cell treatment Carvykti (ciltacabtagene autoleucel) in the UK. 

In March 2022, the US Food and Drug Administration (FDA) granted authorization for Carvykti, a chimeric antigen receptor (CAR) T-cell therapy developed by Johnson & Johnson (J&J) and its partner Legend Biotech, to treat white blood cell cancer.

The treatment was being assessed in the UK by the National Institute of Health and Care Excellence (NICE) in order to make the therapy available on the National Health Service (NHS). However, charity organization Myeloma UK released a statement on Monday stating that J&J has decided to not pursue authorization from NICE at this time.

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Reasons for pushing back the UK launch of Carvykti have not been explicitly specified but Myeloma UK referenced “issues with supply” due to increased global demand. Moreover, the charity highlighted the complex nature of manufacturing CAR-T products and said, “it is believed that it is not going to be possible to produce the supply needed to support a program in the UK.”

The decision to not continue with the consideration for approval from NICE for the treatment to be available on the NHS will not affect clinical trials.

“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,” said Sophie Castell, CEO at Myeloma UK.

“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.”

The charity has said it will be contacting J&J and meeting with various clinicians to establish what can be done advance access to CAR-T cell treatments in the UK.

A spokesperson for J&J remained mute on the potential manufacturing issues detailed in Myeloma UK’s statement, instead telling us “There are multiple clinical, patient, and logistical factors that can impact the production of cilta-cel, which differs from small molecule or biologic oncology therapies. Despite focused efforts, Janssen is not currently in a position to progress with the NICE appraisal process.”

They added: “Janssen does not comment on manufacturing capacity.”

Post-approval patient access

With hefty price tags, investments down 44% from 2021, record layoffs in the space, Big Pharma departures, regulatory agencies struggling to keep up with the speed of innovation, and continued supply chain shortages, it is no surprise that patient access is still challenging post-approval.

Susan Nichols, chief business officer at ViroCell Biologics, told delegates attending Phacilitate’s Advanced Therapies Week in Miami, Florida in January there is “limited patient access post approval [and] sustainable access and reimbursement are the goal.”

Nichols highlighted that in Europe “seven out of the 23 approved therapies have been withdrawn out of the 254 clinical trials that have been initiated this year.” She called for the industry to “make it easier” as the “drugs are only affective when patients receive the benefit, not just a specific geography or socioeconomic subset.”

Update: This article was updated at 12.20 after receiving J&J’s responses.