Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

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Country Product Indication Sponsor Date
3-[3-(6-guanidino-1-oxoisoindolin-2yl) propanamido]-3-(pyridine-3yl) propanoic acid dihydrochloride Retinopathy of prematurity Advanced Imaging Projects, LLC;7947 Brookside Ct.;Lake Worth, Florida, 33467 January 17, 2017
(E)-N’-(1-(5-Chloro-2-hydroxy phenyl)Ethylidene)-3-((4-Methyl Piperazin-1-yl) Sulfonyl) Benzohydrazide Mesylate Ewing's sarcoma Salarius Pharmaceuticals;2450 Holcombe Boulevard, Suite J-608;Houston, Texas, 77021 January 12, 2017
(E)-N�-(1-(5-Chloro-2-hydroxy phenyl)Ethylidene)-3-((4-Methyl Piperazin-1-yl) Sulfonyl) Benzohydrazide Mesylate Ewing's sarcoma Salarius Pharmaceuticals;2450 Holcombe Boulevard, Suite J-608;Houston, Texas, 77021 January 12, 2017
[5,10,15,20-tetrakis(4-carboxyphenyl)-21H,23H-porphine]manganese(III) chloride Cockayne syndrome Institut Pasteur January 12, 2017
3-pentylbenzeneacetic acid sodium salt Alström syndrome ProMetic Pharma SMT Limited January 12, 2017
Antroquinonol Pancreatic Cancer Biological Consulting Europe Ltd January 12, 2017
Anti-interleukin 21 (NNC0114-0006) in combination with liraglutide Type I Diabetes Mellitus with residual beta cell function Novo Nordisk, Inc.;800 Scudders Mill Road, P. O. Box 846;New Jersey, 08536 January 12, 2017
(6aR, 10aR)-3-(1′,1′-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid Systemic scleroderma TMC Pharma Services Ltd January 12, 2017
Autologous dendritic cells incubated ex vivo with zebularine and factor VIII Haemophilia A Idogen AB January 12, 2017
5-aminolevulinic acid Glioma Centre Hospitalier Universitaire de Lille January 12, 2017
Doxorubicin hydrochloride in a lipid-based pegylated nanoparticle modified with a 31-aminoacid peptide targeting nucleolin Malignant pleural mesothelioma TREAT U, S.A. January 12, 2017
Fluticasone propionate Eosinophilic esophagitis Eosinophilic esophagitis Adare Pharmaceuticals srl January 12, 2017
Human donor haematopoietic stem and progenitor cells that have been treated ex vivo with the protein transduction domain of the HIV-1 transactivation protein fused to MYC transcription factor Treatment in haematopoietic stem cell transplantation Coté Orphan Consulting UK Limited January 12, 2017
Genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimized shRNA-insensitive wildtype PABPN1 Oculopharyngeal muscular dystrophy Clinipace GmbH January 12, 2017
Human hepatoma cell line HepaRG in bioartificial liver Acute liver failure Hep-Art Medical Devices BV January 12, 2017
Humanised IgG1 monoclonal antibody against the receptor-binding site of human placental growth factor Medulloblastoma Oncurious NV January 12, 2017
Glyburide Severe cerebral edema in patients with acute ischemic stroke Biogen Chesapeake LLC;225 Binney Street;Cambridge, Massachusetts, 02142 January 12, 2017
Leuprorelin acetate Congenital hypogonadotropic hypogonadism Stichting Centre for Human Drug Research (CHDR) January 12, 2017
Tadekinig alfa Hemophagocytic lymphohistiocytosis AB2 Bio Ltd;EPFL Innovation Park, Building B;Lausanne January 12, 2017
Hydroxychloroquine Antiphospholipid syndrome Centre Hospitalier Universitaire d'Angers January 12, 2017
Pioglitazone hydrochloride Sensorineural hearing loss Regiomedica GmbH January 12, 2017
Pr-D-Cys-Met-Pip-Arg-Leu-Arg-Sar-Cys-Lys-Arg-Pro-Tyr-Tle-Leu-OH Perinatal asphyxia VECT-HORUS January 12, 2017
Methyldopa Type 1 diabetes in DQ8 positive patients with residual beta cell function IM Therapeutics LLC;11001 West 120th Avenue, Suite 400;Broomfield, Colorado, 80021 January 12, 2017
Recombinant adeno-associated viral vector serotype 9 containing the human N-alpha-acetylglucosaminidase gene Mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) Abeona Therapeutics Europe SL January 12, 2017
Recombinant IgG degrading enzyme ofStreptococcus pyogenes Prevention of graft rejection following solid organ transplantation Hansa Medical AB January 12, 2017