For help on how to get the results you want, see our search tips.
329 results
Medicine
Orphan designations Remove Orphan designations filter
Paediatric investigation plans Remove Paediatric investigation plans filter
Direct healthcare professional communication Remove Direct healthcare professional communication filter
Medicine type
Orphan medicine Remove Orphan medicine filter
-
List item
Direct healthcare professional communication (DHPC): Myalepta (metreleptin): inconsistencies in the package leaflet
Active substance: Metreleptin, DHPC type: Quality defect, Last updated: 18/06/2020 -
List item
Direct healthcare professional communication (DHPC): Defitelio (defibrotide): Do not use for prophylaxis of veno-occlusive disease (VOD) after post-hematopoietic stem-cell transplantation (HSCT)
Active substance: defibrotide, DHPC type: Lack of effect, Safety signal, Last updated: 13/06/2022 -
List item
Direct healthcare professional communication (DHPC): Natpar (parathyroid hormone) 100 micrograms/dose powder and solvent for solution for injection: expected shortage from June 30th, 2022
Active substance: parathyroid hormone, DHPC type: Medicine shortage, Last updated: 29/04/2022 -
List item
Direct healthcare professional communication (DHPC): Ocaliva (obeticholic acid): New contraindication for the treatment of primary biliary cholangitis (PBC) in patients with decompensated liver cirrhosis or a history of prior hepatic decompensation
Active substance: Obeticholic acid, DHPC type: New contraindication, Last updated: 09/06/2022 -
List item
Direct healthcare professional communication (DHPC): Strimvelis® (autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase [ADA] cDNA sequence): first case of lymphoid T cell leukaemia after insertional oncogenesis
Active substance: autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human haematopoietic stem/progenitor (CD34+) cells, DHPC type: Adverse event, Last updated: 22/03/2021 -
List item
Direct healthcare professional communication (DHPC): Prevymis (letermovir) concentrate for solution for infusion - Essential to administer through sterile 0.2 micron or 0.22 micron polyethersulfone (PES) in-line filter
Active substance: Letermovir, Last updated: 11/09/2020 -
List item
Direct healthcare professional communication (DHPC): Zolgensma (onasemnogene abeparvovec): risk for thrombotic microangiopathy
Active substance: onasemnogene abeparvovec, DHPC type: Type II variation, Last updated: 18/03/2021 -
List item
Direct healthcare professional communication (DHPC): Polivy (polatuzumab vedotin) 140 mg powder for concentrate for solution for injection: Important information on plastic vial flip-off cap colour
Active substance: polatuzumab vedotin, DHPC type: Quality defect, Last updated: 04/05/2020 -
List item
Orphan designation: Setmelanotide for: Treatment of Bardet-Biedl syndrome (updated)
Date of designation: 21/08/2019, Positive, Last updated: 30/09/2022 -
List item
Orphan designation: Cyclic pyranopterin monophosphate (fosdenopterin) for: Treatment of molybdenum-cofactor deficiency type A (updated)
Date of designation: 20/09/2010, Positive, Last updated: 30/09/2022 -
List item
Orphan designation: Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene for: Treatment of haemophilia A (updated)
Date of designation: 21/03/2016, Positive, Last updated: 28/09/2022 -
List item
Orphan designation: budesonide for: Treatment of primary IgA nephropathy (updated)
Date of designation: 18/11/2016, Positive, Last updated: 14/09/2022 -
List item
Orphan designation: Efgartigimod alfa for: Treatment of myasthenia gravis (updated)
Date of designation: 10/08/2022, Positive, Last updated: 12/09/2022 -
List item
Orphan designation: Asciminib for: Treatment of chronic myeloid leukaemia (updated)
Date of designation: 24/03/2020, Positive, Last updated: 07/09/2022 -
List item
Orphan designation: Sirolimus for: Treatment of chronic non-infectious uveitis
Date of designation: 30/08/2011, Withdrawn, Last updated: 30/08/2022 -
List item
Orphan designation: recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein, Eladocagene exuparvovec for: Treatment of aromatic L-amino acid decarboxylase deficiency
Date of designation: 18/11/2016, Positive, Last updated: 10/08/2022 -
List item
Orphan designation: (S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate for: Treatment of carcinoid syndrome
Date of designation: 30/05/2016, Positive, Last updated: 28/07/2022 -
List item
Orphan designation: Recombinant human acid sphingomyelinase (also known as olipudase alfa) for: Treatment of Niemann-Pick disease
Date of designation: 24/06/2022, Positive, Last updated: 27/07/2022 -
List item
Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) for: Treatment of spinal muscular atrophy
Date of designation: 19/06/2015, Positive, Last updated: 25/07/2022 -
List item
Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy
Date of designation: 13/04/2007, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene for: Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency
Date of designation: 26/08/2005, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of follicular lymphoma
Date of designation: 11/11/2015, Positive, Last updated: 12/07/2022 -
List item
Orphan designation: dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract) for: Treatment of epidermolysis bullosa
Date of designation: 23/02/2011, Positive, Last updated: 04/07/2022 -
List item
Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide for: Treatment of microscopic polyangiitis (avacopan)
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022 -
List item
Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide (avacopan) for: Treatment of granulomatosis with polyangiitis
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022