Ciltacabtagene autoleucel (cilta-cel), a chimeric antigen receptor T-cell (CAR-T) therapy to treat refractory or relapsed myeloma, is being appraised by the Medical Services Advisory Committee (MSAC) for listing on the Medicare Benefits Schedule. 

The Leukaemia Foundation has provided consumer comments in relation to the application for cilta-cel, a B-cell maturation antigen-directed chimeric antigen receptor T-cell to treat refractory or relapsed multiple myeloma.  

The therapy involves taking the patient’s own T-cells (a type of white blood cell that is an essential part of the immune system) from their peripheral blood and genetically modifying them before infusing them back into the patient to treat the myeloma. 

Adult patients with refractory or relapsed myeloma who have received at least three prior lines of therapy (including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody) will be eligible for the therapy.  

The Leukaemia Foundation’s Head of Research, Policy and Advocacy, Dr Peter Diamond said, “the clinical trial data indicates that treatment with cilta-cel in heavily pre-treated patients with myeloma can result in early, deep, and durable responses, with a manageable safety profile”.  

The clinical trial on which the listing application is based had no Australian trial sites, so the Leukaemia Foundation sought insights from three Australian-based clinicians who are involved with a related clinical trial to support our comments.   

“We also sought comments from the Blood Cancer Taskforce which is supportive of the application as proposed,” said Dr Diamond.   

The Leukaemia Foundation observes that patients with relapsed/refractory myeloma have few other treatment options, especially at the third or fourth line.  

“This therapy is a significant advance for these heavily pre-treated patients. Its use may potentially lead to long-term remission for some patients and represents a vast improvement on other currently available therapies beyond third line treatment,” said Dr Diamond. 

Andrew Dodd is one of the first Australians to have CAR T-cell therapy with cilta-cel on a clinical trial. Read his story. 

PBAC update 

And, in latest news, at the March 2022 meeting of the Pharmaceutical Benefits Advisory Committee (PBAC), the following myeloma medications will be considered as new listings or a change to listing:  

• Carfilzomib (Kyprolis®) – a resubmission for use in combination with lenalidomide and dexamethasone, for the treatment of relapsed and or refractory myeloma.  
• Ixazomib (Ninlaro®) – a resubmission for use in combination with lenalidomide and dexamethasone, for the treatment of relapsed and/or refractory myeloma in patients who have received at least two prior therapies. 
• Selinexor (Xpovio®) – for use in combination with dexamethasone, for the treatment of adult patients with relapsed and/or refractory myeloma, who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. 

• Selinexor (Xpovio®) – for use in combination with dexamethasone, for the treatment of relapsed and/or refractory myeloma.