Everything you care about in one place
Follow feeds: blogs, news, RSS and more. An effortless way to read and digest content of your choice.
Get Feederema.europa.eu
Orphan designations
Get the latest updates from Orphan designations directly as they happen.
Follow now 186 followers
Latest posts
Last updated 3 days ago
Orphan designation: Humanised IgG1 monoclonal antibody against human epidermal growth factor receptor 2 Treatment of gastric cancer, 22/05/2025 Positive
3 days ago
Orphan designation: Humanised IgG1 monoclonal antibody against human epidermal growth factor receptor...
Orphan designation: Taladegib Treatment of idiopathic pulmonary fibrosis, 22/05/2025 Positive
3 days ago
Orphan designation: Taladegib Treatment of idiopathic pulmonary fibrosis, 22/05/2025 Positive
Orphan designation: 1-{4-[2-(5-ethoxymethyl-2-methyl-phenylamino)-oxazol-5-yl]-phenyl}-imidazolidin-2-one Treatment of acute myeloid leukaemia, 22/05/2025 Positive
3 days ago
Orphan designation: 1-{4-[2-(5-ethoxymethyl-2-methyl-phenylamino)-oxazol-5-yl]-phenyl}-imidazolidin-2-one Treatment of acute myeloid leukaemia, 22/05/2025 Positive
Orphan designation: Induced pluripotent stem cells-derived myogenic progenitor cells Treatment of Becker muscular dystrophy, 22/05/2025 Positive
3 days ago
Orphan designation: Induced pluripotent stem cells-derived myogenic progenitor cells Treatment of Becker...
Orphan designation: Ramiprilat Treatment of Stargardt's disease, 12/03/2013 Withdrawn
3 days ago
Orphan designation: Ramiprilat Treatment of Stargardt's disease, 12/03/2013 Withdrawn
Orphan designation: Adeno-associated virus serotype C102 containing the human GLA gene Treatment of Fabry disease, 13/04/2022 Withdrawn
3 days ago
Orphan designation: Adeno-associated virus serotype C102 containing the human GLA gene Treatment...
Orphan designation: Radiprodil Treatment of GRIN-related neurodevelopmental disorder, 22/05/2025 Positive
8 days ago
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
8 days ago
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
8 days ago
Orphan designation: Tranilast Treatment of idiopathic pulmonary fibrosis, 22/05/2025 Positive
Orphan designation: florbetaben (18F) Diagnosis of ATTR amyloidosis, 22/05/2025 Positive
8 days ago
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
9 days ago
Orphan designation: Adeno-associated viral vector serotype 2i8 containing the human LAMP2 isoform...
Orphan designation: Equine polyclonal immunoglobulin F(ab')2 fragments against ricin Treatment of ricin poisoning, 22/05/2025 Positive
9 days ago
Orphan designation: Equine polyclonal immunoglobulin F(ab')2 fragments against ricin Treatment of ricin...