The European Medicines Agency (EMA) is set to host an event, the « Multistakeholder Workshop on Patient Registries » on February 12-13, 2024. This two-day hybrid workshop promises to delve into the intricacies of patient registries, offering a unique platform for regulators, registry holders, industry professionals, and a broad spectrum of stakeholders to engage in meaningful discussions.…
EMA is launching a pilot to support the translation of basic research developments into medicines that could make a difference in patients’ lives in the European Economic Area (EEA). The pilot is open to academic sponsors and non-profit organisations who are developing advanced therapy medicinal products (ATMPs). These medicines for human use are based on genes, tissues…
The European Medicines Agency (EMA) has recommended a conditional marketing authorisation in the European Union (EU) for Tecvayli (teclistamab), a monoclonal antibody that targets BCMA and CD3 proteins, for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an…
In the months of May and June 2022, the European Medicines Agency (EMA) recommended granting various forms of marketing authorisation in the European Union (EU) for the following rare disease-targeted therapies: First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder: Xenpozyme (olipudase alfa), a therapy for the treatment of non-central…
The European Medicines Agency (EMA) is organising a joint EMA/HMA/TEHDAS Data Quality Framework for Medicines workshop as a virtual meeting on April 7th from 13.00 – 17.15 CET. This workshop seeks to engage stakeholders early in the drafting process of an EU Network data quality framework, to ensure a robust starting point, in line with their…
In October 2021, the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) announced a pilot project to support the repurposing of medicines. To support not-for-profit organisations and academia ahead of the upcoming deadline for submission (February 28th), the EMA is hosting a « walk-in » clinic webinar on February 17th from 17.00 – 18.00 CET. The webinar will:…
The European Medicines Agency’s (EMA) Clinical Trials Information System (CTIS) has gone live as of January 31st. CTIS is the backbone of the Clinical Trials Regulation that will harmonise the assessment and supervision of clinical trials in the European Union. The Clinical Trials Regulation and CTIS will strengthen clinical trials in the EU, ensuring better…
The European Medicines Agency (EMA) is organising a virtual event on « Research in regulatory science – addressing the needs » to inform the public and stakeholders about regulatory science research needs and how stakeholders can engage with these regulatory science research needs. The webinar will take place on January 18th 2022 from 10.00 – 12.00 CET. Gaps exist in regulatory science that…
The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) are launching a pilot project to support the repurposing of medicines. The aim of this initiative is to support not-for-profit organisations and academia to gather or generate sufficient evidence on the use of an established medicine in a new indication with the view to have this new use formally…
Based on evidence from a single-arm clinical trial, the European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union for the gene therapy SKYSONA™ for the treatment of children with cerebral adrenoleukodystrophy (CALD), a severe form of a rare inherited neurological disease. SKYSONA™, developed by bluebird bio, is a one-time treatment which can…