Orphan designation: H-L-tryphophanyl-L-seryl-glycyl-L-tryptophanyl-L-seryl-L-seryl-L-cysteinyl-L-seryl-L-arginyl-L-seryl-L-cysteinyl-glycyl-OH (disulfide bond), acetate salt Treatment of spinal cord injury, 15/02/2023 Positive

Orphan designation: adeno-associated viral vector serotype 9 expressing fukutin-related protein Treatment of limb girdle muscular dystrophy, 15/02/2023 Positive

Orphan designation: patidistrogene bexoparvovec Treatment of limb girdle muscular dystrophy, 15/02/2023 Positive

Human medicines European public assessment report (EPAR): Mixtard, insulin human (rDNA), Date of authorisation: 07/10/2002, Revision: 20, Status: Authorised

Orphan designation: melatonin Treatment of perinatal asphyxia, 15/02/2023 Positive

Veterinary medicines highlights 2023

Orphan designation: Messenger RNA encoding Cas9, Single guide RNA targeting the human KLKB1 gene Treatment of hereditary angioedema, 11/08/2023 Positive

Veterinary medicines: Highlights of 2023

QRD Appendix V - Adverse-drug-reaction reporting details

Orphan designation: Idronoxil Treatment of soft tissue sarcoma, 22/05/2023 Positive