EMA recommends three new medicines and an orphan treatment for rare Rett Syndrome
EMA’s human medicines committee (CHMP) recommended six medicines for approval at its June 2026 meeting
At its monthly meeting, the human medicines committee approved six medicines, issued three negative opinions and gave positive opinions on the extensions of thirteen therapeutic indications.
CSL Seqirus had a mostly good week. In addition to the Commission starting its investigation into possible disparagement (see below), EMA has recommended granting a marketing authorisation for Aujemflu, a vaccine to protect people aged 50 years and older against influenza.
Catherine Feore
However, the CHMP recommended that Tavneos (avacopan), a medicine used to treat two inflammatory conditions in blood vessels, have its market authorisation revoked. The Committee found that data provided from its main ‘Advocate’ study had been incorrect and misleading. As the study was found to breach good clinical practice (GCP), it was concluded that it could not be relied on for the earlier positive decision.
CSL Seqirus published a letter from their Senior Vice President for Medical Affairs, Dr Deborah Long addressed to “the ANCA /(Anti-neutrophil cytoplasmic antibody) Associated Vasculitis Community”, acknowledging that the news may be difficult and “naturally raises questions about ongoing treatment”. The company advises patients to speak with their treating physician to review their needs.
“We deeply value our well-established partnership with the vasculitis community and will continue to provide further updates to ensure people living with AAV and their healthcare providers are informed and supported through this transition. We remain focused on bringing innovative treatment options to patients living with rare diseases,” wrote Long.
Tavneos was developed by ChemoCentryx, which was acquired by Amgen in 2022. It is commercialized outside the US in selected countries by CSL.
The CHMP also recommended approval of Hopledo (levodopa/carbidopa, Zambon) for the treatment of Parkinson's disease and Onswik (insulin efsitora alfa, Eli Lilly), a once-weekly insulin for the treatment of type 2 diabetes in adults.
Orphan medicines for Rett Syndrome approved
CHMP recommended granting a marketing authorisation for Daybu (trofinetide, Acadia Pharmaceuticals) following a re-examination procedure. Daybu is used to treat the neurobehavioural symptoms of Rett syndrome in adults and children aged 5 years and older. The recommendation follows an initial refusal of the application.
"For decades, families in Europe affected by Rett syndrome have had no medicine specifically approved for the neurobehavioral symptoms of this condition, despite the profound impact they have on almost every aspect of daily life,” said President of Rett Syndrome Europe, Pedro Rocha. “The CHMP’s positive opinion represents hope for thousands of European Union individuals living with this devastating condition, their families and caregivers."
Two biosimilars
Two biosimilars were approved: Denosumab Ascend (prevention of skeletal-related events and treatment of giant cell tumour of bone, denosumab, Ascend GmbH); and, Nylaspeg (reduction of neutropenia in adults, pegfilgrastim, Qilu Pharma Spain S.L.)
Three negative decisions
Three negative decisions were given to: Tacquell (treatment of melanoma, Netherlands Cancer Institute); Xervyteg (treatment of adult patients with acute graft-versus-host disease, allogeneic faecal microbiota, pooled, MaaT PHARMA); and, Yartemlea (haemopoietic stem cell transplant-associated thrombotic microangiopathy, Omeros Ireland Limited).
“We are disappointed by the CHMP’s opinion, particularly given the lethal nature of TA-TMA, the absence of an approved treatment for this condition in Europe, and the totality of the clinical trial and real-world data supporting narsoplimab’s efficacy and safety,” said Chairman and Chief Executive Officer of Omeros, Gregory A. Demopulos. “We believe strongly that YARTEMLEA warrants approval in Europe, just as it received approval in the U.S.”
The company will continue to provide the treatment on compassionate grounds, but says this means that it will only reach a fraction of those who could have been treated had approval been granted.