The first targeted therapy for AAV has been approved in Australia, representing an important advancement for many patients living with the rare disease.
ANCA-associated vasculitis (AAV) patients in Australia will now be able to access the oral small-molecule C5a receptor inhibitor avacopan.
The TGA has approved avacopan (Tavneos, Vifor) in combination with a rituximab or cyclophosphamide regimen for the treatment of granulomatosis polyangiitis (GPA) and microscopic polyangiitis (MPA), the two main forms of AAV.
“This is an exciting step forward for patients with AAV in Australia, to have a new class of medication approved for use,” said Adelaide-based rheumatologist Dr Joanna Tieu.
Avacopan’s approval was supported by the results of the ADVOCATE trial, published in the New England Journal of Medicine. The randomised, double-blind, active-controlled, phase III trial in 331 patients with AAV took place in 20 countries, including Australia.
Participants were randomised to receive either rituximab or cyclophosphamide (followed by azathioprine/mycophenolate) and either avacopan or prednisone. Patients in both groups who experienced worsening symptoms could be treated with glucocorticoid rescue therapy.
“Really, it’s about reducing steroid burden to patients,” Dr Tieu told Rheumatology Republic.
“The hope [with the ADVOCATE trial] was that it would reduce steroid burden on the patient, and the patients taking avacopan ended up with a very low dose of steroid compared with what we’d consider standard therapy now,” she said.
The ADVOCATE study met its primary endpoints of disease remission at 26 weeks and sustained remission at 52 weeks, demonstrating superiority to a prednisone-based standard of care with respect to sustained remission at 52 weeks.
The most common adverse effects were nausea, headache, hypertension, diarrhoea, vomiting, rash, fatigue, upper abdominal pain, dizziness, blood creatinine increase and paraesthesia. A three-year early access program involving 30 patients reported no new or elevated safety signals.
The drug was approved by the FDA in October 2021 and has also been approved in Japan, Europe, the UK and Canada.
“This long-awaited new class of medication represents an important advancement for many patients living with AAV”, clinical immunologist Dr Daman Langguth said in a press release.
“We are confident that this novel therapy will provide more options to help control the disease, extend remission and meet major unmet medical needs in the treatment of AAV,” said Dr Langguth, who’s based at Wesley Hospital in Queensland and is also director of immunology at Sullivan Nicolaides Pathology.
The company has confirmed it will pursue a PBS listing.
“We are committed to bringing Tavneos (avacopan) to Australian patients living with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) and plan to make a submission to the Pharmaceutical Benefits Advisory Committee as soon as possible, reinforcing our ongoing commitment to improving the care of patients living with these rare diseases with significant unmet medical need,” James McDonnell, the general manager of CSL Vifor Australia and New Zealand, told Rheumatology Republic.
In the meantime, said Mr McDonnell, an access program for Tavneos was currently operating in Australia for patients who meet the approved indication.
“CSL Vifor reviews all requests for access to its approved medications that are not yet reimbursed in Australia on a case-by-case basis, in accordance with TGA Guidelines, internal policies and the Medicines Australia Code of Conduct,” he said.
Physicians who have a patient who may require access can contact the company through compassionate_AU@viforpharma.com.
“I really look forward to the results of the PBAC application to make this widely available in Australia,” Dr Tieu said.
