Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-10-14 | autologous mononuclear cells derived from human cord blood | BrainRepair UG (Germany) | neonatal encephalopathy |
Granting of the orphan status in the EU |
2016-10-14 | carbamazepine | University of Newcastle upon Tyne (UK) | metaphyseal chondrodysplasia, Schmid type |
Granting of the orphan status in the EU |
2016-10-14 | chemically modified human recombinant sulfamidase | Swedish Orphan Biovitrum (Sobi) (Sweden) | mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) |
Granting of the orphan status in the EU |
2016-10-14 | crenolanib besylate | Arog Pharmaceuticals (USA - TX) | soft tissue sarcoma |
Granting of the orphan status in the EU |
2016-10-14 | crenolanib besylate | Arog Pharmaceuticals (USA - TX) | acute myeloid leukaemia |
Granting of the orphan status in the EU |
2016-10-14 | cxendin (9-39) | Eiger BioPharmaceuticals (USA - CA) | noninsulinoma pancreatogenous hypoglycaemia syndrome |
Granting of the orphan status in the EU |
2016-10-14 | fenretinide | Clinipace (Germany) | peripheral T-cell lymphoma |
Granting of the orphan status in the EU |
2016-10-14 | hematopoietic stem cells modified with a lentiviral vector containing the CD18 gene | Centro de Investigación Biomédica en Red (CIBER) (Spain) | leukocyte adhesion deficiency type I |
Granting of the orphan status in the EU |
2016-10-14 | melatonin | Therapicon (Italy) | Smith-Magenis syndrome |
Granting of the orphan status in the EU |
2016-10-14 | P-ethoxy growth factor receptor-bound protein 2 antisense oligonucleotide | Clinical Network Services (UK) | acute myeloid leukaemia |
Granting of the orphan status in the EU |
2016-10-14 | self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene | Ser-mes Planificación SL (Spain) | mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) |
Granting of the orphan status in the EU |
2016-10-14 | tadekinig alfa | Coté Orphan Consulting UK (UK) | hemophagocytic lymphohistiocytosis (HLH) |
Granting of the orphan status in the EU |
2016-10-14 | tetrofosmin | ProActina (Greece) | glioma |
Granting of the orphan status in the EU |
2016-10-14 | ubiquinol | Centro de InvestigaciónBiomédica en Red (CIBER) (Spain) | primary coenzyme Q10 deficiency syndrome |
Granting of the orphan status in the EU |
2016-12-08 | [5,10,15,20-tetrakis(4-carboxyphenyl)-21H,23H-porphine]manganese(III) chloride | Institut Pasteur (France) | Cockayne syndrome |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | 5-aminolevulinic acid | Centre Hospitalier Universitaire de Lille (France) | glioma |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | hydroxychloroquine | Centre Hospitalier Universitaire d'Angers (France) | antiphospholipid syndrome |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | leuprorelin acetate | Stichting Centrefor Human Drug Research (CHDR) (The Netherlands) | congenital hypogonadotropic hypogonadism |
Positive opinion for the granting of the orphan status in the EU |
2016-12-08 | fluticasone propionate | Adare Pharmaceuticals (USA - NJ) | eosinophilic oesophagitis |
Positive opinion for the granting of the orphan status in the EU |
2017-04-11 | pioglitazone hydrochloride | Regiomedica (Germany) Strekin (Switzerland) | sudden sensorineural hearing loss | Granting of the orphan status in the EU |
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