TAVNEOS Approved in Japan for ANCA-Associated Vasculitis

Sometimes, certain drugs may be approved in different countries and not in others. However, drug approval spanning multiple countries highlights the importance of getting drugs and treatments into the hands of those that need them. According to Pharmafile, TAVNEOS (avacopan) was recently approved in Japan. Altogether, this treatment is designed for patients with ANCA-associated vasculitis, or, in particular, microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA).


So what exactly is TAVNEOS and why are we excited about its approval? According to a separate news release published by Yahoo News, TAVNEOS is:

a first-in-class, orally-administered small molecule that employs a novel, highly targeted mode of action in complement-driven autoimmune and inflammatory diseases. By precisely blocking the receptor (the C5aR) for the pro-inflammatory complement system fragment known as C5a on destructive inflammatory cells such as blood neutrophils, avacopan arrests the ability of those cells to do damage in response to C5a activation, which is known to be the driver of ANCA-associated vasculitis.

The recent approval hinged on data from the Phase 3 ADVOCATE clinical trial. Altogether, 331 patients with either MPA or GPA enrolled. During the trial, patients received TAVNEOS treatment, which was compared to the current standards-of-care. Researchers determined that TAVNEOS shows benefits in reducing symptoms and improving patient outcomes.

Beyond ANCA-associated vasculitis, the drug developers are also exploring TAVNEOS as a potential therapeutic option for patients with lupus nephritis, C3 glomerulopathy (C3G), and hidradenitis suppurativa.

Microscopic Polyangiitis (MPA)

As described above, ANCA-associated vasculitis actually includes multiple categories; outside of the two already listed, ANCA-associated vasculitis also includes eosinophilic granulomatosis with polyangiitis. For the purpose of this article, I’m going to focus on microscopic polyangiitis (MPA). However, if you’d like a more comprehensive understanding of ANCA-associated vasculitis as a whole, head to this helpful primer.

Microscopic polyangiitis is a rare disorder characterized by blood vessel inflammation (vasculitis). Doctors are not sure exactly what causes MPA, though an overactive or abnormal immune response could play a role. In patients with MPA, small blood vessels within the nervous system, skin, lungs, joints, and kidneys are often affected. As these blood vessels become inflamed, organs can become damaged, causing dysfunction or failure. Symptoms vary based on where in the body is affected. Some symptoms may include:

  • Unintended weight loss
  • Skin lesions or rashes
  • Fatigue
  • Muscle and joint pain
  • Lowered mobility
  • Shortness of breath and/or difficulty breathing
  • Swollen legs
  • Coughing up blood
  • Appetite loss
  • Hematuria (bloody urine)
  • Fever
  • Nerve damage
  • Respiratory failure
  • Kidney failure
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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