EU regulators moved to revoke a rare-disease drug over its pivotal trial data
The CHMP's June meeting recommended pulling avacopan from the EU market on data-integrity grounds, refused three cell and biological therapies, and reversed itself on a Rett syndrome drug it had earlier rejected.
Most European Medicines Agency committee meetings are read for what they approve. The June meeting of the Committee for Medicinal Products for Human Use is worth reading for what it withdrew.
The CHMP finalised its review of Tavneos (avacopan) and recommended that the medicine's marketing authorisation in the EU be revoked, on the grounds that its benefits are no longer proven to outweigh its risks [s1]. Avacopan is used in adults with severe, active granulomatosis with polyangiitis or microscopic polyangiitis — two rare inflammatory conditions of the blood vessels [s1].
The reason given is unusual. The review was initiated to assess new information that raised questions regarding the data integrity of the main study supporting the medicine's marketing authorisation in the EU [s1].
That is a different category of regulatory problem from the ones that normally trigger a withdrawal. A safety signal means the harms turned out larger than expected. A failed confirmatory trial means the benefit did not replicate. A data-integrity question means the regulator is no longer confident about what the original evidence showed at all — and for a drug authorised on a single pivotal study, there is nothing left underneath it.
A CHMP recommendation is not the final word. Opinions go to the European Commission, which issues the legally binding decision. Until that happens, the authorisation stands.
Three refusals in one meeting
The committee adopted negative opinions for three medicines [s1], an unusually high number for a single sitting, and the three have something in common: all are complex biological or cell-based products.
Tacquell — autologous melanoma-derived tumour infiltrating lymphocytes, expanded outside the body — was refused for the treatment of advanced melanoma [s1]. Xervyteg, a pooled allogeneic faecal microbiota product, was refused for acute graft-versus-host disease, the condition in which donor cells from a bone marrow or stem cell transplant attack the recipient's body shortly after transplant [s1]. And Yartemlea (narsoplimab) was refused for transplant-associated thrombotic microangiopathy in adults and children from two years of age, a serious and potentially life-threatening complication of haematopoietic stem cell transplantation [s1].
Each of these targets a condition with limited options and a small, seriously ill population — exactly the setting where regulators face the most pressure to accept imperfect evidence, and exactly where single-arm or small trials are hardest to interpret. The EMA publishes question-and-answer documents explaining each refusal [s1].
And one reversal
The committee also went the other way on a product it had previously rejected. Following a re-examination, it recommended granting a marketing authorisation for Daybu (trofinetide), for the neurobehavioural symptoms of Rett syndrome in adults and paediatric patients aged five years and older — symptoms that include repetitive hand movements, restlessness, general mood problems, anxiety, and sleep and communication problems [s1].
The CHMP had initially refused the application [s1]. After re-examination it recommended that authorisation could be granted, but for a restricted indication [s1]. Re-examination is the applicant's formal appeal route within the CHMP process; reversals happen, but a narrowed indication is the usual price.
A referral over an inactive ingredient
Separately, the committee started a review of Rifadin 20 mg/ml oral suspension and syrup, a rifampicin-containing antibiotic used to treat tuberculosis and other serious infections [s1]. The concern is not the antibiotic. It is the level of one of the excipients, diethanolamine, which has been classified as a possible carcinogen on the basis of studies in rodents exposed over long periods to very high doses [s1].
The review was initiated at the request of the Dutch medicines agency under Article 31 of Directive 2001/83/EC [s1]. The rodent-dose caveat in the EMA's own description is important context: a possible-carcinogen classification derived from high-dose animal exposure does not establish risk at the levels present in a medicine, which is precisely what the referral exists to determine. The oral suspension and syrup formulations are the paediatric-friendly ones for a drug that is a cornerstone of tuberculosis treatment.
What was approved
The committee recommended six new medicines for approval and another 12 for extension of their therapeutic indications [s1].
The new approvals include Aujemflu, an inactivated influenza vaccine for people aged 50 and older; Hopledo (levodopa/carbidopa) for adults with Parkinson's disease and moderate-to-severe motor fluctuations who have not been sufficiently stabilised on oral levodopa-based regimens; and Onswik (insulin efsitora alfa) for type 2 diabetes in adults [s1]. Two biosimilars also received positive opinions: Denosumab Ascend and Nylaspeg (pegfilgrastim) [s1].
Two indication changes are worth flagging for anyone tracking vaccine policy. The committee recommended lowering the age of administration for Imvanex — the vaccine used against smallpox, mpox and vaccinia — from 12 years and older to two years and older [s1]. And it recommended limiting the use of Ixchiq, the live chikungunya vaccine, to people aged 12 and older who are at high risk of acquiring chikungunya infection [s1]. One widens access; the other narrows it.
The Mounjaro decision
A quieter item may matter most commercially. The CHMP finalised its assessment of an application to extend the use of Mounjaro (tirzepatide) to reduce the risk of serious cardiovascular events in adults with type 2 diabetes who already have cardiovascular disease [s1].
The committee did not recommend granting the new indication [s1]. It did agree to include the relevant data submitted with the application in the medicine's product information, so that healthcare professionals have access to up-to-date data on the drug's effects in that population [s1].
That is a middle outcome that European regulators use more often than is generally appreciated: the evidence goes into the label without becoming a claim the manufacturer can market.
Two applications to extend the use of the Spiromax budesonide-and-formoterol inhalers as a reliever-only treatment for mild asthma were withdrawn [s1].
What to watch
The avacopan case is the one with the longest tail. If the Commission follows the recommendation, patients currently treated will need a transition plan, and other regulators that authorised the drug on the same pivotal study will have to decide whether to open their own reviews.
Sources
- [s1] Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 June 2026 — European Medicines Agency, 26 June 2026. https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-june-2026
Sources
- Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 June 2026 — European Medicines Agency , June 26, 2026
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