New PBS listing of targeted therapy for rare cancer announced

Pharma News: A next-generation targeted therapy has been listed on the Pharmaceutical Benefits Scheme (PBS) for the treatment of Waldenstrom Macroglobulinemia (WM), a rare form of blood cancer. This is the first targeted therapy to be listed on the PBS for the treatment of WM, marking a significant milestone for these patients, who up until now, had no other treatment choice but chemotherapy.

As of July 1, the PBS listing of BeiGene’s oral BRUKINSA (zanubritinib) will provide eligible patients affordable access.

“For most people it’s a huge shock to be diagnosed with a very rare disease, particularly with an unpronounceable name,” said David Young, leader of local patient advocacy group WMozzies. “The prospect of having a life-limiting condition can be frightening, as patients with WM are often older and don’t cope well with harsh chemotherapy, which is all that’s been available. We’ve been working tirelessly for years to have the first WM targeted treatment on the PBS, and we’re thrilled for the WM community that this is now a reality.”

“This is a momentous day for Australian medical research and for our patients. My colleagues and myself were the first doctors in the world to treat patients with BRUKINSA in clinical trials, and it gives me great personal satisfaction to see this potentially life-changing drug now widely available to patients with WM.” said Professor Con Tam, Head of Lymphoma Service at Alfred Health, and Professor of Haematology at Monash University.

“Patients with WM often suffer poor quality of life from their cancer and from the side-effects of chemotherapy, so we hope the availability of BRUKINSA will help to greatly relieve this suffering.”

Arthur Alston, Head of Medical Affairs, APAC, BeiGene “Today represents a significant step forward on BeiGene’s journey to ensuring patients have access to innovative medicines at an affordable price, regardless of where they live. People living with WM are a historically underserved community facing considerable unmet need here in Australia. Our hope is that BeiGene will also be able to positively impact other rare diseases that have not been given the attention they deserve”.

Adrian Dunstan, General Manager, BeiGene Australia and New Zealand: “The first WM patient in a global BRUKINSA trial was treated here in Australia, which has been a priority location for many of our clinical trials. Now BRUKINSA is approved in 50 markets worldwide across numerous blood cancer indications. We’re excited about the prospect of this therapy as well as our general progress as we become industry leaders in the blood cancer space.”

BRUKINSA was approved by the TGA for the treatment of mantle cell lymphoma, another rare form of blood cancer, in October 2021, and listed on the PBS in March 2022.