Targeted Drugs for Rare Autoimmune Disease Hit Regulatory Milestones

Medical anca associated vasculitis avacopan

New, more targeted treatments are on the horizon for the rare autoimmune disease ANCA-associated vasculitis, with a nod towards European approval for a first-in-class drug and positive phase II results from a rival treatment.

In November, the Swiss firm Vifor Fresenius Medical Care Renal Pharma (VFMCRP) received a thumbs-up from the EMA for avacopan, a first-in-class drug for the rare condition ANCA-associated vasculitis. The European Commission is expected to make a final market approval decision for avacopan in the first quarter of next year. 

Avacopan was approved for the treatment of ANCA-associated vasculitis in the US in October, where VFMCRP’s partner ChemoCentryx will market avacopan under the brand name Tavneos. 

ANCA-associated vasculitis is an umbrella term for a group of rare, progressive autoimmune diseases where the immune system attacks small blood vessels. The condition stems from overactivation of the complement system, one of the immune system’s first lines of defense. Elevated levels of the inflammatory protein C5a in particular are believed to play an important role in the autoimmune disease, which can lead to life-threatening organ dysfunction such as kidney failure or lung damage. 

The current standard of care includes long-term steroid medication to suppress the whole immune system. However, these drugs carry substantial clinical risks for bone health and fertility as well as increasing the risk of infections, diabetes, hypertension, cataracts, and adrenal insufficiency. Avacopan is designed to selectively block the action of C5a and produce fewer adverse effects.

There is a significant need for new and innovative therapies to help patients with this systemic and debilitating condition to have better outcomes, a better quality of life, and to reduce the challenging side effects of current standard of care,” said Vifor Pharma Global Head of Communications, Nathalie Ponnier.

We are confident that Tavneos has the potential to become the new standard for the treatment for patients suffering from ANCA-associated vasculitis.”

The FDA approval and positive opinion from the EMA label avacopan for use in combination with standard medications for the two main forms of ANCA-associated vasculitis: severe, active granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). The drug has also been given the green light by Japan’s Ministry of Health and Labor Welfare for the treatment of patients with GPA or MPA.

ChemoCentryx is now discussing with regulators how to move the drug forward for the treatment of the rare kidney disease C3 glomerulopathy (C3G) and skin condition hidradenitis suppurativa. The firm is also planning to initiate clinical development of the drug for the kidney disease lupus nephritis.

Avacopan is not alone in the race to find alternative treatments and reduce steroid use for ANCA-associated vasculitis. Last month, German biotech InflaRx showcased promising results for treatment of the disease in a phase II study of its anti-C5a antibody vilobelimab. InflaRx’s drug proved as effective as a standard dose of glucocorticoids in the 57 recruited patients.

Our recently reported trial data showed that vilobelimab treatment induced a high rate of clinical remission in relapsing or newly diagnosed ANCA-associated vasculitis patients, while considerably reducing the use of corticosteroids and corticosteroid-related toxicity,” InflaRx’s CEO, Niels Riedemann, told me.

The emerging treatments may be timely during the Covid-19 pandemic. The symptoms of ANCA-associated vasculitis and Covid-19 often resemble each other, making diagnosis a challenge. And a few early-stage reports speculate that, in very rare cases, Covid-19 infections and even mRNA vaccinations could trigger or exacerbate ANCA-associated vasculitis.

Richard Kitching, kidney and vasculitis specialist at Monash Health and Monash University, said there were several ways to improve the future management of ANCA-associated vasculitis. These include tailoring treatments to the individual and developing new treatments targeting the immune system.

Kitching — whose laboratory has received financial support from Vifor Pharma and Visterra — also pointed to the importance of new treatments that target pathways that are more specific to ANCA-associated vasculitis.

The interactions between C5a and its receptor are an example,” he told me. “Neutralizing this pathway has a good biological rationale and treatments that target C5a-C5aR may at least partially replace glucocorticoids.”

The replacement of glucocorticoids with a more targeted therapy could in the future be viewed as a milestone in the treatment of ANCA-associated vasculitis. However, more study and time is needed to more fully define and understand the place of these new treatments in ANCA-associated vasculitis. We don’t yet understand their best use in different phases of the disease.”

2 December 2021: Nathalie Ponnier affiliation corrected to Vifor Pharmarnrn rnrnCover image via Elena Resko

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