Approval Application for ANCA-Associated Vasculitis Drug Amended by Developer

According to a story from ANCA Vasculitis News, the biopharmaceutical company ChemoCentryx has recently made an amendment to its approval application for its investigational therapy. This therapy, called avacopan, is being developed for ANCA-associated vasculitis, a group of autoinflammatory disorders. The amended application was submitted as a result of recommendations laid out during a meeting between the US Food and Drug Administration (FDA) and an independent advisory committee of medical experts.

About ANCA-Associated Vasculitis

ANCA-associated vasculitis is a group of diseases that are characterized by the damage and destruction of blood vessels as a result of inflammatory activity. The disease is associated with the presence of anti-neutrophil cytoplasmic antibodies (ANCA). These are autoantibodies that target antigens present on neutrophils (the most common type of white blood cell) and monocytes. This means that the mechanism of the disease is autoimmune, in which the body’s own immune system mistakenly attacks healthy body tissue. Symptoms of ANCA associated-vasculitis include kidney inflammation, fever, weight loss, abdominal pain, bloody stools, purpura, nose bleeds, muscle pain, arthritis, bloody cough, vision problems, headaches, stroke, heart attack, and high blood pressure. Treatment of the disease is primarily focused on controlling inflammation and suppressing immune system activity. Common medications include cyclophosphamide, rituximab, and prednisone. Antibiotics may be necessary in cases of infection. To learn more about ANCA associated-vasculitis, click here.

A Divided Vote

The vote tallies from the meeting suggest that there was some controversy surrounding the avacopan data. In a 10-8 vote, the committee concluded that the drug’s safety profile was sufficient for approval. The committee also agreed that the risk-benefit profile was acceptable for the dose level being called for in the application, but the committee was still divided along a 10-8 split. Meanwhile, there was a 9-9 tie vote on whether the efficacy data for the drug supported approval.

These close votes were far from a ringing endorsement for the original application. As a result, ChemoCentryx will be submitting additional data in front of the FDA in hopes of addressing the concerns raised by the committee. In the phase 3 trial upon which the application was based, treatment with avacopan was determined to be non-inferior to treatment with the glucocorticoid prednisone, and avacopan also showed superior remission rates after 52 weeks. However, the committee felt this may not be sufficient since many participants were treated with glucocorticoids outside of the trial protocol.

Avacopan is also capable of boosting glucocorticoid exposure, potentially throwing another wrench into the findings. The company pointed out recently that the data illustrated that the drug was better at restoring kidney function, particularly in patients with more serious kidney disease. A decision on the approval is expected on October 7th.

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