ChemoCentryx nabs FDA approval of Tavneos for ANCA-associated vasculitis

ChemoCentryx said Friday that the FDA cleared Tavneos (avacopan) as an adjunctive treatment for adults with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis, sending the company's shares up as much as 80%. The decision makes Tavneos the first orally-administered complement C5a inhibitor approved in the US.

Specifically, Tavneos is authorised to treat the two main forms of the disease – granulomatosis with polyangiitis and microscopic polyangiitis – in combination with standard therapy. CEO Thomas Schall indicated that the drug would be "available to clinicians and patients in the next few weeks," with the expected annual wholesale acquisition cost set at between$150,000 and $200,000, which ChemoCentryx noted is at the upper end of the price for orphan drugs.

The company's application was backed by results from the Phase III ADVOCATE trial, which randomised 330 patients to receive Tavneos or prednisone, both on top of treatment with rituximab or cyclophosphamide/azathioprine. The study met its primary endpoints of disease remission at 26 weeks and sustained remission at 52 weeks, according to findings published in the NEJM earlier this year.

More data filed after AdCom

ChemoCentryx's stock took a major hit in May after an FDA advisory panel was split about the drug's efficacy and safety profile, raising questions about how the ADVOCATE trial was designed and suggesting there may be a need for more liver toxicity testing. However, the panel ultimately voted by a 10-8 margin that the benefit-risk profile was good enough for approval (see ViewPoints: ChemoCentryx completes painful roundtrip).

The FDA was originally expected to make its decision in July, and Raymond James analyst Steven Seedhouse, who has estimated peak US sales of $1.9 billion for the drug, said it was a "pure coin flip" which way the agency would go. However, ChemoCentryx later submitted additional data for its filing to address points raised at the advisory meeting, pushing the action date to this month.

Tavneos was cleared in Japan for ANCA-associated vasculitis late in September, while the drug is also under development for the treatment of patients with C3 glomerulopathy, hidradenitis suppurativa and lupus nephritis. ChemoCentryx owns commercial rights to Tavneos in the US, while Vifor Pharma has exclusive rights to commercialise the drug in markets outside of the country.

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