There is a high unmet need for therapies for patients with AL amyloidosis patients and daratumumab (Darzalex®) subcutaneous (SC) is the first ever therapy to be approved in Australia for AL amyloidosis.

At its intracycle meeting in May 2022, the Pharmaceutical Benefits Advisory Committee (PBAC) recommended daratumumab for use in combination with cyclophosphamide, bortezomib and dexamethasone (CyBorD) for newly diagnosed systemic AL amyloidosis.

The PBAC recognised there were no treatments on the Pharmaceutical Benefits Scheme (PBS) specifically for this condition and it considered that the addition of daratumumab SC with CyBorD offered a high therapeutic value.

The Leukaemia Foundation provided comments on behalf of consumers in support of the application when it was first submitted to PBAC, for its November 2021 meeting.

Having researched this medication, the patient noted “how it had helped those with AL who have come out of remission”, and expressed a wish to “have the opportunity to have this drug from the onset of my diagnosis… To catch it early with effective drugs is my best chance and I see this drug as one that can assist me greatly.” And this patient, whose amyloidosis had been diagnosed early, added, “without the best drugs being available to me, it is of no real advantage knowing this if I cannot afford the best treatment for this disease. Being made available to me and others in my situation will give hope and relief to people who are told they have a life-threatening rare disease”.

The Leukaemia Foundation also sought commentary from clinicians who had experience with daratumumab therapy for Australians with AL amyloidosis, and they noted that although survival data is currently too immature for comment, treatment with daratumumab is associated with improved haematologic response, improved heart and kidney organ responses, and less relapse.

From trial results to date, the Leukaemia Foundation understands that daratumumab used in this combination is associated with additional side effects, but these are similar to existing treatments and are manageable. Clinicians we spoke to also noted quality of life improvements, and similar results to the international Phase III ANDROMEDA* trial.

Light chain (AL) amyloidosis is a progressive and rare condition, and the prognosis is very poor. There are no Therapeutic Goods Administration-approved treatments or treatments listed under the Pharmaceutical Benefits Scheme (PBS) for this disease in Australia.

The Leukaemia Foundation urged the PBAC to recommend daratumumab for listing on the PBS for eligible patients given the high unmet need for therapies for patients with AL amyloidosis patients, the acceptable safety profile of daratumumab, and the evidence of overall survival benefit.

* Results of the ANDROMEDA trial showed that adding the CD38-targeted therapy, daratumumab, to bortezomib-containing therapy improves outcomes for people with newly diagnosed light-chain (AL) amyloidosis. In the Phase III clinical trial, that was available at several Australian centres, daratumumab therapy increased complete response rates and survival free from major organ deterioration or haematologic progression. The results were published in the New England Journal of Medicine in July 2021. Study co-author, Dr Simon Gibbs, wrote on his Twitter feed @simondjgibbs, “Very pleased and excited to see our hard work published in #NEJM - confirming daratumumab as the new standard of care for treatment of AL Amyloidosis. Already approved by the FDA, let’s hope it’s approved soon for Australian patients.”