MHRA Reviews Avacopan Licence Over Trial Data Issues
In its latest notice, the Medicines and Healthcare products Regulatory Agency, or MHRA, said it is reviewing the benefits and risks of avacopan, sold as Avacopan Vifor and previously known as Tavneos. The review was triggered by information that raises questions about the integrity and reliability of data from the pivotal clinical study that helped support the medicine's UK licence. That matters because avacopan is used in serious, rare autoimmune illnesses. In the UK, it is authorised in combination with a rituximab or cyclophosphamide regimen for adults with severe, active granulomatosis with polyangiitis, known as GPA, or microscopic polyangiitis, known as MPA.
If those names are unfamiliar, you are not alone. GPA and MPA are rare conditions in which the immune system attacks small blood vessels, which can damage organs and make treatment decisions especially important. That is why a review like this can sound alarming even before any final decision has been made. It is also worth slowing down here. The MHRA has not said patients should stop avacopan. It has said it is checking whether the new information changes what the key study showed about how well the medicine works, and whether the overall benefit-risk balance still supports its use.
When regulators talk about a medicine's benefit-risk balance, they are asking a plain question: do the expected benefits still outweigh the known and possible harms for the people who take it? A medicine can have side effects and still be approved if the evidence shows that, for the right patients, it does more good than harm. What this means in plain English is that the review is not only about whether the medicine has risks. It is about whether the evidence behind its benefits can still be trusted in the same way. If the study data behind an approval becomes uncertain, the regulator has to revisit the judgement it made when the licence was granted.
The legal basis matters too. The MHRA said the review is being carried out under section 68 of the Human Medicines Regulations 2012. In simple terms, that gives the regulator the power to revoke, vary or suspend a UK marketing authorisation if the application, or the material supplied with it, is incorrect, or no longer supports a positive benefit-risk balance. For readers who do not follow drug regulation closely, a marketing authorisation is the formal permission that allows a medicine to be sold for specific uses. It is not a one-off stamp that can never be questioned. It can be revisited when fresh evidence or fresh concerns appear.
For now, the existing safety advice stays in place. The MHRA said the current risk minimisation guidance continues to apply while the review is under way. According to the product information on medicines.org.uk, that includes monitoring liver function and white blood cell count, as well as assessing patients for any serious infections. That point is easy to miss, but it matters. A review does not leave doctors and patients with no guidance. Healthcare professionals are being reminded to weigh the individual benefits and risks for each patient, using the advice that is already in the official product information.
The clearest message for patients is also the simplest one: do not stop treatment without speaking to your healthcare professional. For people living with severe, active GPA or MPA, treatment choices are rarely straightforward, and a decision that is right for one patient may not be right for another. If you are taking avacopan and feel worried by this news, the safest next step is to ask the clinician managing your care what the review means for you personally. That conversation is usually more useful than making a sudden change based on a headline.
The MHRA says it will publish the outcome once the review is complete, including any new advice for healthcare professionals and patients. In the meantime, suspected adverse reactions can still be reported through the MHRA Yellow Card scheme, which helps the regulator build a fuller picture of how medicines are working in real life. There is a wider lesson here as well. A medicine review is not proof that a treatment has failed, and it is not proof that everything is fine. It is the system doing what it is meant to do: checking the evidence again when important questions are raised, and making sure patients and clinicians are told what to do next.