Orphan designation: Radiprodil Treatment of GRIN-related neurodevelopmental disorder, 22/05/2025 Positive
Orphan designation: Doxorubicin,Liposomal,Pegylated Treatment of soft-tissue sarcoma, 22/05/2025 Positive
Orphan designation: Tranilast Treatment of idiopathic pulmonary fibrosis, 22/05/2025 Positive
Orphan designation: florbetaben (18F) Diagnosis of ATTR amyloidosis, 22/05/2025 Positive
Orphan designation: Adeno-associated viral vector serotype 2i8 containing the human LAMP2 isoform B transgene Treatment of Danon disease, 22/05/2025 Positive
Orphan designation: Equine polyclonal immunoglobulin F(ab')2 fragments against ricin Treatment of ricin poisoning, 22/05/2025 Positive
Orphan designation: Tertomotide Treatment of progressive supranuclear palsy, 22/05/2025 Positive
Orphan designation: recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1 Treatment of calciphylaxis, 22/05/2025 Positive
EMEA-001222-PIP01-11-M01
Opinion/decision on a Paediatric investigation plan (PIP): Constella, linaclotide, decision type: PM: decision on the application for modification of an agreed PIP, therapeutic area: Gastrointestinal disorders, PIP number: P/0529/2022