The European Medicines Agency has begun a formal review of Tavneos (avacopan) after emerging information raised questions about the integrity of key clinical trial data supporting its EU authorisation, with potential implications for adults living with rare autoimmune vasculitis.
Following the 11 December 2025 political agreement, EU institutions are moving towards formal adoption of a new Directive and Regulation that will reshape how medicines—including orphan and paediatric medicines—are developed, authorised and supplied across the European Union.
Open for consultation from 12 December to 12 April, this draft ICH guideline sets out how to design and submit robust patient preference studies to inform regulatory decision‑making across a medicine’s lifecycle.
Gathering key stakeholders, including EU policymakers, industry leaders, patient advocacy groups, researchers, and healthcare providers. Its goal is to identify concrete actions and incentives to enhance innovation across the rare disease ecosystem.
With over 300 million people affected by rare diseases, timely access to effective therapies is critical. Despite strong alignment in regulatory expectations, patients in many regions face years of delays highlighting the need for improved international coordination.
