Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-11-18 | brincidofovir | Chimerix UK (UK) | smallpox |
Granting of the orphan status in the EU |
2016-11-18 | human monoclonal antibody against activin | Regeneron Pharmaceuticals (USA - NY) | fibrodysplasia ossificans progressiva |
Granting of the orphan status in the EU |
2016-11-18 | L-selenomethionine | Université de Montpellier (France) | facioscapulohumeral muscular dystrophy |
Granting of the orphan status in the EU |
2016-11-18 | particles comprised of methacrylic acid based co-polymer, cross-linked with a bi-functional crosslinker,purified to bind L-phenylalanine and L-phenylalanine containing peptides | MipSalus (Denmark) | hyperphenylalaninaemia |
Granting of the orphan status in the EU |
2016-11-18 | recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic Lamino acid decarboxylase protein | Voisin Consulting (France)/Agilis Biotherapeutics | aromatic L-amino acid decarboxylase deficiency/AADC Deficiency |
Granting of the orphan status in the EU |
2016-11-18 | sodium benzoate | Lucane Pharma (France) | argininosuccinic aciduria |
Granting of the orphan status in the EU |
2016-11-18 | sodium benzoate | Lucane Pharma (France) | N-acetylglutamate synthase deficiency |
Granting of the orphan status in the EU |
2016-11-18 | Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Gly-Gly-Asp-Leu-Leu-Pro-Arg-Gly-Ser | Dr Ulrich Granzer (Germany) | Huntington’s disease |
Granting of the orphan status in the EU |
2016-11-18 | zinc gluconate | Université de Montpellier (France) | facioscapulohumeral muscular dystrophy |
Granting of the orphan status in the EU |
2016-11-18 | human monoclonal antibody against activin | Regeneron Pharmaceuticals (USA - NY) | fibrodysplasia ossificans progressiva |
Granting of the orphan status in the EU |
2016-12-12 | recombinant human monoclonal IgG1 antibody against programmed death ligand-1 (anti-PD-L1) | Merck Serono (Germany) | gastric cancer |
Granting of the orphan status in the EU |
2016-12-12 | adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes | FondazioneTelethon (Italy) | retinitis pigmentosa |
Granting of the orphan status in the EU |
2016-12-12 | arsenic trioxide capsule (oral) | Medsenic (France) | graft-versus-host disease |
Granting of the orphan status in the EU |
2016-12-12 | cabiralizumab | Albany Regulatory Consulting (UK) | tenosynovial giant cell tumour, localised and diffuse type |
Granting of the orphan status in the EU |
2016-12-12 | propranolol | The Anticancer Fund (Belgium) | soft tissue sarcoma | Granting of the orphan status in the EU |
2016-12-12 | 68Ga-DOTA-pABzA-DIG-dPhe-Gln-Trp-Ala-Val-Gly-His-NHCH[(CH2-CH(CH3)2]2 | Advanced Accelerator Applications (France) | diagnosis of gastrointestinal stromal tumours |
Granting of the orphan status in the EU |
2016-12-12 | adeno-associated viral vector serotype 8 containing the human CNGA3 gene under the control of a cone arrestin promoter | Universitätsklinikum Tübingen (Germany) | achromatopsia caused by mutations in the CNGA3 gene |
Granting of the orphan status in the EU |
2016-12-12 | dantrolene sodium | Alan Boyd Consultants Ltd (UK) | Wolfram syndrome. |
Granting of the orphan status in the EU |
2016-12-12 | ibudilast | MediciNova (USA - CA) | amyotrophic lateral sclerosis |
Granting of the orphan status in the EU |
2017-12-26 | ivosidenib | QRC Consultants (UK) - Agios Pharmaceuticals (USA - MA) |
|
Submission of a Market Application in the US |
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