MHRA Completes Thorough Review of Avacopan Vifor: Key Findings on Benefit-Risk Balance Revealed

The Medicines and Healthcare products Regulatory Agency (MHRA) has concluded its assessment of Avacopan Vifor, previously branded as Tavneos, and has determined that the evidence currently available does not support a favourable benefit-risk balance for the medication.

This review was initiated following concerns regarding the integrity and reliability of data derived from the pivotal clinical trial that was essential for the medicine’s authorisation. After a thorough evaluation of the evidence and in consultation with the Commission on Human Medicines (CHM), along with input from patients and healthcare professionals, the MHRA has reached the conclusion that the clinical study can no longer be regarded as a reliable source for demonstrating the efficacy of Avacopan Vifor.

Suspension of Use and Supply

In light of these findings, the MHRA has notified the marketing authorisation holder of its decision to suspend the use, supply, and sale of Avacopan Vifor to new patients effective from 1 September 2026. This action is taken to safeguard patient welfare, allowing those currently receiving the medication to transition safely to alternative treatments.

During a six-month managed withdrawal period, supply will continue for existing patients, after which the MHRA plans to revoke the UK marketing authorisation on 1 March 2027. The marketing authorisation holder has assured that there will be sufficient supply to support patients throughout this transition.

Regulatory Framework and Indications

This decision falls under Regulations 69 and 71 of the Human Medicines Regulations 2012, which facilitate the suspension of the use, supply, and sale of medicinal products. Avacopan Vifor, when used in conjunction with a regimen of rituximab or cyclophosphamide, was indicated in the UK for the treatment of adults suffering from severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), both of which are rare autoimmune diseases that affect small blood vessels.

Julian Beach, Executive Director of Healthcare Quality and Access at the MHRA, emphasised: “Patient safety is our highest priority. Following a thorough review of the available evidence, we have concluded that the data supporting the efficacy of Avacopan Vifor is no longer sufficiently reliable to support a positive benefit-risk balance. We have therefore taken regulatory action to suspend the use, supply, and sale of this medicine to new patients. We intend to subsequently revoke the medicine’s UK marketing authorisation following a transitional period.”

Guidance for Healthcare Professionals and Patients

Healthcare professionals are advised against initiating treatment with Avacopan Vifor for new patients and should promptly review the cases of those already receiving the medication, considering suitable alternative treatment options as necessary.

Patients currently taking Avacopan Vifor are strongly encouraged not to cease treatment without first consulting their specialist healthcare provider. Existing risk minimisation advice remains applicable, which includes regular monitoring of liver function, with an increased frequency of recommended testing, as well as monitoring white blood cell counts and assessing for any serious infections.

Any suspected adverse reactions should be reported through the MHRA Yellow Card scheme to ensure ongoing safety monitoring.

Background on Avacopan Vifor

Avacopan Vifor, which was authorised in the UK on 6 May 2022, was intended for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). The MHRA oversees the regulation of all medicines and medical devices in the UK, ensuring their efficacy and safety through robust, evidence-based assessments.

The MHRA operates as an executive agency of the Department of Health and Social Care, while the Commission on Human Medicines (CHM) serves as the government’s independent expert scientific advisory body.

For media inquiries, please reach out via email to newscentre@mhra.gov.uk or call 020 3080 7651.