Orphan designation: telitacicept Treatment of myasthenia gravis, 18/07/2025 Positive
Orphan designation: telitacicept Treatment of myasthenia gravis, 18/07/2025 Positive
Orphan designation: telitacicept Treatment of myasthenia gravis, 18/07/2025 Positive
Orphan designation: curcumin Treatment of Charcot-Marie-Tooth disease, 18/07/2025 Positive
Orphan designation: ribavirin Treatment of hepatitis E, 18/07/2025 Positive
Human medicines European public assessment report (EPAR): Triumeq, abacavir,dolutegravir,lamivudine, Date of authorisation: 31/08/2014, Revision: 36, Status: Authorised
Orphan designation: avutometinib,defactinib Treatment of ovarian cancer, 18/07/2025 Positive
Orphan designation: palmitoyl-conjugated tricyclo-DNA antisense oligonucleotide 5'-Palm-C6-*GGA GAT GgC AGT TTC-3 Treatment of Duchenne muscular dystrophy, 18/07/2025 Positive
Orphan designation: luminol monosodium Treatment of Duchenne muscular dystrophy, 18/07/2025 Positive
Academia
Orphan designation: human bispecific monoclonal antibody targeting ALK1 and BMPRII Treatment of hereditary haemorrhagic telangiectasia, 18/07/2025 Positive
Orphan designation: adeno-associated viral vector serotype 9 containing the human CTNNB1 gene Treatment of autosomal dominant polycystic kidney disease, 18/07/2025 Positive