Orphan designation: sonlicromanol hydrochloride Treatment of inherited mitochondrial oxidative phosphorylation defects, 18/07/2025 Positive

4 September 2025

Orphan designation: Ivosidenib Treatment of myelodysplastic syndromes, 18/07/2025 Positive

4 September 2025

Orphan designation: telitacicept Treatment of myasthenia gravis, 18/07/2025 Positive

4 September 2025

Orphan designation: curcumin Treatment of Charcot-Marie-Tooth disease, 18/07/2025 Positive

4 September 2025

Orphan designation: ribavirin Treatment of hepatitis E, 18/07/2025 Positive

4 September 2025

Human medicines European public assessment report (EPAR): Triumeq, abacavir,dolutegravir,lamivudine, Date of authorisation: 31/08/2014, Revision: 36, Status: Authorised

4 September 2025

Orphan designation: avutometinib,defactinib Treatment of ovarian cancer, 18/07/2025 Positive

4 September 2025

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

4 September 2025

Orphan designation: luminol monosodium Treatment of Duchenne muscular dystrophy, 18/07/2025 Positive

4 September 2025

Academia

4 September 2025