Orphan designation: epertinib Treatment of amyotrophic lateral sclerosis, 26/02/2025 Positive
Orphan designation: Autologous CD34+ cell enriched population containing haematopoietic stem and progenitor cells transduced ex vivo with a lentiviral vector encoding the human ADA2 gene Treatment of adenosine deaminase 2 deficiency (DADA2), 27/02/2025 Positive
Human medicines European public assessment report (EPAR): Emcitate, tiratricol, Date of authorisation: 12/02/2025, Status: Authorised
Orphan designation: adeno-associated virus sector serotype rh74 containing the human SGCG gene Treatment of limb-girdle muscular dystrophy, 27/02/2025 Positive
Cancer Medicines Forum: December 2024, Online, European Medicines Agency, Amsterdam, the Netherlands, from 16 December 2024, 13:00 (CET) to 16 December 2024, 15:00 (CET)
Presentation - Optimizing the dosage of human prescription drugs and biological products for the treatment of oncologic diseases: FDA guidance (Mirat Shah)
Minutes of the Cancer Medicines Forum - December 2024
Read our press release from 10 March on ECIM 2025 on Medscape by Cristina Ferrario:
Human variation electronic application form (eAF) training session and Q&A clinic, Online, European Medicines Agency, Amsterdam, the Netherlands, from 8 November 2024, 10:00 (CET) to 8 November 2024, 11:00 (CET)
Q&A clinic Human variations web-based electronic Application Form (eAF) for non-CAPs, Online, European Medicines Agency, Amsterdam, the Netherlands, from 25 October 2024, 10:00 (CEST) to 25 October 2024, 10:30 (CEST)