MHRA: Avacopan Vifor UK Suspension for New Patients Announced

• The MHRA has decided to suspend the use, supply, and sale of Avacopan Vifor to new patients in the UK from September 1, 2026.
• This action stems from concerns regarding the integrity and reliability of data from the pivotal clinical study, leading to a conclusion that the medicine's benefit-risk balance is no longer positive.
• The UK marketing authorisation for Avacopan Vifor is intended to be revoked on March 1, 2027, following a six-month managed withdrawal period for existing patients.
• Healthcare professionals should not initiate new treatments, and existing patients should not stop Avacopan Vifor without specialist consultation.

The MHRA's decisive action regarding the Avacopan Vifor UK suspension has been taken under the authority granted by Regulations 69 and 71 of the Human Medicines Regulations 2012.

The Medicines and Healthcare products Regulatory Agency (MHRA) has concluded its comprehensive review of Avacopan Vifor, previously known as Tavneos, leading to a significant regulatory decision regarding its availability in the UK. The agency has determined that the evidence currently available no longer supports a positive balance between the medicine's benefits and risks. Consequently, the MHRA has informed the marketing authorisation holder of its decision to suspend the use, supply, and sale of Avacopan Vifor to new patients, effective from September 1, 2026.

This suspension is a precursor to the intended revocation of the UK marketing authorisation for Avacopan Vifor, slated for March 1, 2027. To ensure a safe transition for individuals currently undergoing treatment, a six-month managed withdrawal period will be implemented. During this time, from September 1, 2026, until March 1, 2027, supply of the medicine will be permitted exclusively for existing patients, with the marketing authorisation holder guaranteeing its availability throughout this transitional phase. Avacopan Vifor, which received UK authorisation on May 6, 2022, is indicated for the treatment of adults diagnosed with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), rare autoimmune conditions affecting small blood vessels, when used in combination with a rituximab or cyclophosphamide regimen.

The MHRA's review was initiated following the emergence of information that cast doubt on the integrity and reliability of data derived from the pivotal clinical study that originally underpinned the medicine's authorisation. This raised serious questions about the foundational evidence supporting Avacopan Vifor's efficacy. Following a thorough and detailed assessment of all pertinent evidence, and after receiving expert advice from the Commission on Human Medicines (CHM), the MHRA ultimately concluded that the study can no longer be considered reliable enough to demonstrate the medicine's effectiveness.

The agency also considered representations made by patients and healthcare professionals throughout its review process. The MHRA emphasized that patient safety remains its paramount concern in all regulatory decisions. The conclusion reached was that the data supporting the efficacy of Avacopan Vifor is no longer sufficiently reliable to maintain a positive benefit-risk balance, necessitating the regulatory action taken.

In light of the impending suspension, healthcare professionals have been advised not to initiate treatment with Avacopan Vifor for any new patients. For individuals currently receiving the medicine, healthcare providers are urged to review their care at the earliest opportunity, considering appropriate alternative treatment options where necessary to ensure continuity of effective therapy.

Patients who are presently taking Avacopan Vifor are strongly cautioned against discontinuing their treatment without first consulting their specialist healthcare professional. Existing risk minimisation advice, detailed in the product information, continues to apply. This includes vigilant monitoring of liver function, for which the recommended testing frequency was recently increased, as well as regular monitoring of white blood cell count and assessing patients for any serious infections. Any suspected adverse reactions experienced by patients should be promptly reported through the MHRA Yellow Card scheme.

The MHRA's decisive action regarding the Avacopan Vifor UK suspension has been taken under the authority granted by Regulations 69 and 71 of the Human Medicines Regulations 2012. These specific regulations empower the agency to suspend the use, supply, and sale of a medicinal product when deemed necessary to protect public health. As the primary body responsible for regulating all medicines and medical devices within the UK, the MHRA's mandate is to ensure that these products are both effective and acceptably safe for public use.

This regulatory intervention underscores the MHRA's stringent enforcement capabilities and its commitment to upholding the highest standards of data integrity in pharmaceutical product approvals. The decision to suspend and subsequently revoke a marketing authorisation based on concerns about the reliability of pivotal clinical trial data establishes a clear precedent for robust regulatory oversight. It highlights the critical importance of robust data governance and validation processes throughout the lifecycle of a medicinal product, from initial clinical trials through to post-market surveillance.

This MHRA action establishes a precedent for stringent regulatory enforcement against pharmaceutical products where clinical trial data integrity is compromised, requiring legal and compliance teams to review internal data validation processes and assess regulatory risks for existing and pipeline marketing authorisations in the UK. Pharmaceutical companies must ensure robust data governance to avoid similar product withdrawals.

Source: Original reporting via UK government regulatory body.