orphan designation
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Removal of the EMA orphan designation upon request of the sponsor: cui prodest?

European Journal of Clinical Pharmacology ◽

10.1007/s00228-021-03096-y ◽

2021 ◽

Author(s):

Nicola Montanaro ◽

Giulia Bonaldo ◽

Domenico Motola

Keyword(s):

Orphan Designation

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PRO66 Trends in Withdrawal of Orphan Designation for Marketed Products: EU VS. US and JAPAN

Value in Health ◽

10.1016/j.jval.2020.08.1802 ◽

2020 ◽

Vol 23 ◽

pp. S701

Author(s):

W. Colasante ◽

R. Lawlor ◽

C. Jones ◽

Y. Chen

Keyword(s):

Orphan Designation

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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues (givosiran), Treatment of acute hepatic porphyria

Case Medical Research ◽

10.31525/cmr-2b5a1a4 ◽

2020 ◽

Author(s):

Keyword(s):

Aminolevulinic Acid ◽

Orphan Designation ◽

Covalently Linked ◽

Sirna Oligonucleotide

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Orphan designation: Yttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1, Treatment of pancreatic cancer

Case Medical Research ◽

10.31525/cmr-2b1b7f2 ◽

2020 ◽

Author(s):

Keyword(s):

Pancreatic Cancer ◽

Monoclonal Antibody ◽

Mucin 1 ◽

Orphan Designation ◽

Humanized Monoclonal Antibody

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Orphan designation: Veltuzumab, Treatment of chronic lymphocytic leukaemia

Case Medical Research ◽

10.31525/cmr-2b1b808 ◽

2020 ◽

Author(s):

Keyword(s):

Chronic Lymphocytic Leukaemia ◽

Lymphocytic Leukaemia ◽

Orphan Designation

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Orphan designation: Adeno-associated viral vector serotype 8 containing the human factor-VII gene, Treatment of congenital factor VII deficiency

Case Medical Research ◽

10.31525/cmr-2b06459 ◽

2020 ◽

Author(s):

Keyword(s):

Human Factor ◽

Viral Vector ◽

Orphan Designation ◽

Factor Vii Deficiency ◽

Gene Treatment ◽

Congenital Factor

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Orphan designation: 3,4-diaminopyridine phosphate (amifampridine), Treatment of Lambert-Eaton myasthenic syndrome

Case Medical Research ◽

10.31525/cmr-2af10a4 ◽

2020 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Myasthenic Syndrome

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Orphan designation: dimethyl fumarate, Treatment of bullous pemphigoid

Case Medical Research ◽

10.31525/cmr-2ac6959 ◽

2020 ◽

Author(s):

Keyword(s):

Bullous Pemphigoid ◽

Dimethyl Fumarate ◽

Orphan Designation

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Orphan designation: Human platelet antigen-1a immunoglobulin, Prevention of fetal and neonatal alloimmune thrombocytopenia

Case Medical Research ◽

10.31525/cmr-2ac6957 ◽

2020 ◽

Author(s):

Keyword(s):

Human Platelet ◽

Orphan Designation ◽

Platelet Antigen ◽

Human Platelet Antigen ◽

Alloimmune Thrombocytopenia

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Orphan designation: Efgartigimod alfa, Treatment of immune thrombocytopenia

Case Medical Research ◽

10.31525/cmr-2a47e36 ◽

2020 ◽

Author(s):

Keyword(s):

Immune Thrombocytopenia ◽

Orphan Designation

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