The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended revoking the European Union marketing authorisation for Tavneos (avacopan), citing concerns over the integrity of the clinical trial data that supported the medicine’s approval.

Tavneos is currently used in combination with standard therapies to treat adults with severe, Active Granulomatosis with Polyangiitis (GPA) and Microscopic Polyangiitis (MPA), two rare autoimmune diseases that cause inflammation of blood vessels.

The review was initiated after new information raised concerns about the reliability of data from the pivotal Phase 3 Advocate study, which formed the basis for the medicine’s approval in the EU. Following a comprehensive assessment, the CHMP concluded that the trial had been conducted in breach of Good Clinical Practice (GCP) standards and that the submitted data were inaccurate and misleading, making them unsuitable for demonstrating the medicine’s effectiveness.

The committee also determined that supportive post-marketing evidence and additional analyses of the Advocate study were insufficient to confirm the clinical benefits of Tavneos. As a result, it concluded that the medicine’s benefits could no longer be considered to outweigh its risks.

The recommendation will now be forwarded to the European Commission, which will make the final legally binding decision applicable across all EU Member States. If adopted, Tavneos will no longer be authorised for marketing in the European Union.

The CHMP has advised that no new patients should begin treatment with Tavneos. Healthcare providers have also been instructed to transition patients currently receiving the therapy to appropriate alternative treatments.

The committee highlighted ongoing safety concerns associated with Tavneos, including an increased risk of Drug-Induced Liver Injury (DILI) and Vanishing Bile Duct Syndrome (VBDS), a rare but serious condition that can lead to progressive destruction of the liver’s bile ducts and has been linked to fatal outcomes.

To minimise these risks, the EMA has recommended enhanced liver function monitoring for patients discontinuing the medicine. Patients who have received Tavneos for less than three months should undergo liver function tests at least every two weeks until three months have elapsed since treatment initiation. Those treated for more than three months should continue monitoring every four weeks for up to six months, followed by further assessments as clinically required. Tavneos should be discontinued immediately if VBDS is suspected.

The EMA said a Direct Healthcare Professional Communication (DHPC) outlining these recommendations will be issued to healthcare professionals responsible for prescribing, dispensing or administering the medicine.

Tavneos contains the active substance avacopan and was authorised in the EU for use alongside rituximab or cyclophosphamide-based treatment regimens in patients with severe GPA or MPA. The review was initiated at the request of the European Commission under Article 20 of Regulation (EC) No 726/2004 and included input from the Pharmacovigilance Risk Assessment Committee (PRAC), patient representatives and healthcare professionals.