Orphan designation: antisense oligonucleotide against SYNGAP1 regulatory RNA Treatment of SYNGAP1-related disorder, 20/04/2026 Positive
Orphan designation: upadacitinib Treatment of systemic sclerosis, 20/04/2026 Positive
Orphan designation: coagulation factor V Treatment of congenital factor V deficiency, 20/04/2026 Positive
Procurement and grants
Orphan designation: Momelotinib dihydrochloride monohydrate Treatment of VEXAS syndrome, 20/04/2026 Positive
Human medicines European public assessment report (EPAR): Volibris, ambrisentan, Date of authorisation: 21/04/2008, Revision: 27, Status: Authorised
Orphan designation: oremepermin alfa Treatment of spinal cord injury, 20/04/2026 Positive
Orphan designation: lisaftoclax Treatment of myelodysplastic syndromes, 13/04/2026 Positive
Orphan designation: acetylleucine Treatment of CACNA1A disorders, 25/03/2026 Positive
Orphan designation: exaluren sulfate Treatment of Alport syndrome, 25/03/2026 Positive