The European Medicines Agency has recommended revoking the marketing authorisation of Tavneos (avacopan), concluding that the medicine’s benefits are no longer proven to outweigh its risks after a review found the pivotal clinical trial data to be unreliable.
Tavneos is used to treat adults with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), two rare ANCA-associated vasculitides that cause inflammation of small blood vessels. It was approved in the EU as part of combination therapy with rituximab or cyclophosphamide.
The review was triggered by new information questioning the integrity of the Advocate study, a 331-patient trial that compared a 52-week course of Tavneos against a 20-week course of high-dose corticosteroids, both added to standard treatment. At the time of initial evaluation, the data showed Tavneos was at least as effective as corticosteroids in inducing remission and produced higher 52-week remission rates.
After examining the totality of the available data, the CHMP concluded the Advocate study was conducted in breach of good clinical practice (GCP) principles. Study data submitted during the marketing authorisation assessment were found to be incorrect and misleading and can no longer be relied upon to demonstrate efficacy. Supportive post-marketing data and post-hoc analyses were not considered sufficient to fill the gap.
The decision is particularly consequential because Tavneos carries known risks of drug-induced liver injury (DILI) and vanishing bile duct syndrome (VBDS), a rare condition in which the small bile ducts inside the liver are progressively destroyed. Cases with fatal outcomes have been reported. Without reliable evidence of benefit, these risks become unacceptable.
If the European Commission confirms the recommendation, Tavneos will no longer be authorised in the EU. In the meantime, no new patients should start treatment. Current patients should be switched to suitable alternatives, and liver function must be closely monitored until treatment is permanently stopped, at least every two weeks for patients in their first three months of treatment, and every four weeks for those treated longer, for up to six months. If VBDS is suspected, Tavneos must be discontinued immediately.
The revocation marks a rare post-marketing withdrawal driven not by a new safety signal alone, but by the erosion of the efficacy evidence base itself, a finding that strikes at the foundation of the medicine’s original approval.
Source: EMA News
