Orphan designation: Reldesemtiv Treatment of spinal muscular atrophy, 28/06/2019 Positive
Orphan designation: Reldesemtiv Treatment of amyotrophic lateral sclerosis, 28/02/2020 Positive
Orphan designation: Enzastaurin hydrochloride Treatment of Ehlers-Danlos syndrome, 24/02/2022 Positive
Orphan designation: Cyclo(-gamma-aminobutyryl-L-phenylalanyl-L-tryptophanyl-D-tryptophanyl-L-lysyl-L-threonyl-L phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt Treatment of acromegaly, 06/12/2012 Positive
Orphan designation: Brincidofovir Prevention of cytomegalovirus disease, 28/04/2016 Positive
Orphan designation: Brincidofovir Treatment of adenovirus infection in immunocompromised patients, 14/07/2016 Positive
Orphan designation: autologous CD4+ and CD8+ T cells transduced with lentiviral vector containing an affinity-enhanced T-cell receptor targeting the New York esophageal antigen-1 Treatment of soft tissue sarcoma, 14/07/2016 Positive
PRIME – Paving the way for promising medicines for patients - Factsheet
Handling of competing interests: revised rules for committee members and experts
EMA policy on handling of competing interests of scientific committee members and experts: Draft for public consultation