MHRA Reviews Avacopan After Trial Data Concerns

On 5 June 2026, the MHRA said it had started reviewing avacopan, a medicine used alongside rituximab or cyclophosphamide for adults with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). Those conditions are rare forms of vasculitis, where the immune system attacks small blood vessels and can seriously affect organs including the lungs and kidneys. (gov.uk) As of Monday 8 June 2026, the public MHRA position is that the review is under way and that the outcome will be announced once the work is complete. That matters because avacopan is used in severe autoimmune disease and as part of a broader treatment plan, not as a stand-alone routine medicine. (gov.uk)

The trigger is not a new public recall or an instruction for everyone to stop treatment. The MHRA says it received information raising questions about the integrity and reliability of data from the pivotal clinical study that supported the medicine's licence approval, and it is now checking whether that changes what the study showed about effectiveness and the overall balance of benefit and risk. (gov.uk) **What this means:** when regulators talk about data integrity, they are asking whether the records behind a key study are trustworthy enough to support the original result. That is our plain-English reading of the MHRA notice. In avacopan's case, NICE guidance published in September 2022 had relied on trial evidence suggesting that, after a year, avacopan with standard care was better than standard care alone at keeping disease from getting worse and might reduce steroid toxicity. The current review is testing whether that earlier picture still stands. (gov.uk)

If you're wondering how a medicine can be approved and then reviewed later, this is a useful reminder of how regulation works. A medicine licence is based on a judgement that the benefits outweigh the risks, but that judgement can be revisited if new information appears. MHRA guidance says UK-wide marketing authorisation applications are assessed through national procedures, and the legal framework allows action after approval as well as before it. (gov.uk) Under regulation 68 of the Human Medicines Regulations 2012, the licensing authority may revoke, vary or suspend a UK marketing authorisation if, among other reasons, the product is harmful, its positive therapeutic effects no longer outweigh its risks, it lacks therapeutic efficacy, or the application or material supplied with it is incorrect. So this review is not a paperwork exercise. It is a formal check on whether the licence still rests on solid evidence. (legislation.gov.uk)

For now, the MHRA has not told patients to stop avacopan. It says the existing risk-minimisation advice in the product information stays in place while the review continues. According to the current Summary of Product Characteristics on the electronic medicines compendium, that includes monitoring liver function and white blood cell count, and assessing patients for serious infections. (gov.uk) The current product information goes further than the short MHRA notice. It says white blood cell counts should be checked before treatment starts and monitored as clinically indicated, and that treatment must be temporarily stopped if a patient develops an active, serious infection or certain blood-count or liver-test abnormalities. That does not decide the review's outcome, but it does show why close follow-up matters while the regulator checks the evidence. (medicines.org.uk)

If you're taking avacopan, the clearest advice is also the easiest to miss: do not stop treatment on your own. The MHRA says patients should not stop without speaking to a healthcare professional, and that remains the safest route while the review is open. (gov.uk) A good conversation with your specialist team now is not about panic; it is about context. Ask what the review means for your own illness, what monitoring is due, and which symptoms should prompt urgent advice. The current product information tells patients to report signs of infection, unexpected bruising or bleeding, and symptoms such as swelling of the face, lips or tongue, throat tightness, or breathing difficulty. Anyone can also report a suspected problem through the Yellow Card scheme, but Yellow Card is not a substitute for medical advice. (medicines.org.uk)

For clinicians, the MHRA's message is cautious rather than dramatic. It is reminding healthcare professionals to weigh individual patient benefits and risks carefully during the review, not announcing a blanket stop across the board. As of 8 June 2026, NICE guidance TA825 also remains published and recommends avacopan, within its marketing authorisation, as an option with rituximab or cyclophosphamide when provided under the commercial arrangement. (gov.uk) **What it means for practice:** there is a difference between an ongoing evidence review and a final regulatory decision. Right now, clinicians are being asked to keep current safeguards in place, use judgement case by case, and continue reporting suspected adverse reactions so the national safety picture stays as complete as possible. (gov.uk)

There is a wider media-literacy lesson here as well. A regulatory review is not the same thing as proof that a medicine never worked, and it is not the same thing as a clean bill of health either. It means the evidence is being checked because the standard for keeping a medicine on the market is that it must still be supported by reliable data and an acceptable balance of benefit and risk. That final sentence is our summary of the legal test set out in the MHRA notice and the Human Medicines Regulations. (gov.uk) The next step is straightforward, even if the science is not: the MHRA says it will publish the outcome of the review, along with any new advice for professionals and patients, once the work is complete. Until then, the calmest reading is also the most accurate one: there has been no MHRA instruction for patients to stop treatment across the board, the current monitoring advice still applies, and the key question is whether the main study evidence can still support the licence. (gov.uk)