Orphan designation: extract from Cannabis flower, containing high levels of cannabidiolic acid and <0.3% of tetrahydrocannabinol, Extraction solvent: olive oil, virgin Treatment of Rett syndrome, 25/03/2025 Positive

Orphan designation: allopurinol Treatment of Marfan syndrome, 25/03/2025 Positive

Orphan designation: adeno-associated virus vector serotype 8 containing the human F9 gene Treatment of haemophilia B, 25/03/2025 Positive

Thirteenth meeting of the industry stakeholder platform on research and development support, European Medicines Agency, Amsterdam, the Netherlands, 2 December 2024

Highlight report - 13th Industry stakeholder platform on research and development support

SMS guidance for external users

PSUSA/00002942/202406

Human medicines European public assessment report (EPAR): Ajovy, fremanezumab, Date of authorisation: 28/03/2019, Revision: 13, Status: Authorised

Human medicines European public assessment report (EPAR): SomaKit TOC, edotreotide, Date of authorisation: 08/12/2016, Revision: 21, Status: Authorised

Orphan designation: bexmarilimab Treatment of myelodysplastic syndromes, 25/03/2025 Positive