Orphan designation: adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene Treatment of Duchenne muscular dystrophy, 29/08/2016 Withdrawn

Orphan designation: RNA editing antisense oligonucleotide against the Z mutation of the human SERPINA1 mRNA transcript, sodium salt Treatment of alpha-1 antitrypsin deficiency, 22/08/2025 Withdrawn

Start of procedure: Type II Variation - Extension of indication under evaluation by the CHMP (27 February 2026 - 26 March 2026)

Start of procedure: Type II Variation - Extension of indication under evaluation by the CHMP (30 January 2026 - 26 February 2026)

Questions and answers - Practical arrangements on the companion diagnostics consultation procedure to the European Medicines Agency by notified bodies

Questions and answers - Practical arrangements on the companion diagnostics consultation procedure to the European Medicines Agency by notified bodies - tracked changes

Human medicines European public assessment report (EPAR): Besponsa, inotuzumab ozogamicin, Date of authorisation: 28/06/2017, Revision: 12, Status: Authorised

Human medicines European public assessment report (EPAR): Zercepac, trastuzumab, Date of authorisation: 27/07/2020, Revision: 15, Status: Authorised

Human medicines European public assessment report (EPAR): Imfinzi, durvalumab, Date of authorisation: 21/09/2018, Revision: 28, Status: Authorised

Highlights from the third expert panels and notified bodies workshop