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
Palm tocotrienol complex CADASIL Hovid Berhad;121 Jalan Tunku Abdul Rahman;Ipoh June 13, 2017
Active calcium sensing receptor antagonist derived from tetrahydropyrazolopyrimidine Pulmonary Arterial Hypertension Takeda Development Centre Europe, Ltd.;61 Aldwych;London June 13, 2017
Nandrolone decanoate Duchenne muscular dystrophy Sarcomed AB;25 St�rtloppsv�gen;H�gersten-Liljeholmen June 13, 2017
Humanized monoclonal antibody against human PD-1 Neuroendocrine tumors Novartis Pharmaceuticals Corporation;One Health Plaza, Bldg. 135-566G;East Hanover, New Jersey, 07936 June 8, 2017
Humanized monoclonal immunoglobulin G1 antibody directed against human CD19 conjugated to SG3199 through a protease cleavable valine-alanine linker Diffuse Large B-cell Lymphoma ADC Therapeutics SA;Route de la Corniche, 3B;Epalinges June 8, 2017
Humanized monoclonal immunoglobulin G1 antibody directed against human CD19 conjugated to SG3199 through a protease cleavable valine-alanine linker Mantle cell lymphoma ADC Therapeutics SA;Route de la Corniche, 3B;Epalinges June 8, 2017
Oleoylethanolamide Prader-Willi syndrome NutriForward, LLC;1312 Brown Trail, Suite E;Bedford, Texas, 76022 June 8, 2017
Sodium trans-[tetrachlorobis(1H-indazole)ruthenate(III)] Pancreatic Cancer Intezyne Technologies, Inc.;3720 Spectrum Boulevard, Suite 104;Tampa, Florida, 33612 June 8, 2017
2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-a]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile Idiopathic pulmonary fibrosis Galapagos NV;Generaal de Wittelaan, L11 A#;Mechelen June 8, 2017
20% Lipid emulsion solution Local anesthetic systemic toxicity ResQ Pharma;621 Rose Street;Lincoln, Nebraska, 68502 June 8, 2017
4�-(p-fluorobenzyl)tylosin A Onchocerciasis AbbVie Inc.;Regulatory Affairs, 1 Waukegan Road;North Chicago, Illinois, 60044 June 8, 2017
Recombinant human fibroblast growth factor receptor 3 Achondroplasia TherAchon AG;36 Aeschenvorstadt;Basel June 2, 2017
Autologous Epstein-Barr specific T-cells Post-transplant lymphoproliferative disorder Cell Medica, Inc.;7505 Fannin Street, Suite 200;Houston, Texas, 77054 May 31, 2017
Etelcalcetide Pediatric hyperparathyroidism Amgen, Inc.;1 Amgen Center Drive;Thousand Oaks, California, 91320 May 31, 2017
L-3,4-Dihydroxy-6-[18F]fluorophenylalanine Aromatic amino acid decarboxylase deficiency Advanced Imaging Projects, LLC;7947 Brookside Ct.;Lake Worth, Florida, 33467 May 31, 2017
Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the human COL7A1 gene. Dystrophic epidermolysis bullosa Abeona Therapeutics;6555 Carnegie Blvd, Suite 401;Cleveland, Ohio, 44103 May 24, 2017
Irinotecan liposome injection Small cell lung cancer Ipsen Bioscience, Inc.;650 East Kendall Street;Cambridge, Massachusetts, 02142 May 24, 2017
Resiquimod Cutaneous T-cell lymphoma Galderma Research and Development, LLC;14501 North Freeway;Fort Worth, Texas, 76177 May 24, 2017
Chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p Cutaneous T-cell lymphoma Miragen Therapeutics Europe Ltd May 22, 2017
Poly(oxy-1,2-ethanediyl), .alpha.-hydro-.omega.-hydroxy-,15,15′-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-.alpha.-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain Paroxysmal nocturnal hemoglobinuria Best Regulatory Consulting Ltd May 22, 2017
Recombinant adeno-associated viral vector serotype 6 encoding the B-domain-deleted human factor VIII Haemophilia A Coté Orphan Consulting UK Limited May 22, 2017
Recombinant adeno-associated virus serotype 9 vector expressing codon-optimized miniaturized version of DMD gene Duchenne muscular dystrophy Bamboo Therapeutics, w wholly owned subsidiary of Pfizer;Worldwide Safety & Regulatory, Pfizer Inc.;Groton, Connecticut, 06340 May 22, 2017
Bictegravir/emtricitabine/tenofovir disoproxil fumarate Human Immunodeficiency Virus type 1 infection in pediatric patients Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 May 17, 2017
Sirolimus Tuberous sclerosis NobelPharma Co., LTD;12-10 Nihonbashi-kobunacho;Chuo-ku May 17, 2017
Exenatide Idiopathic intracranial hypertension Alan Boyd Consultants Limited;Electra House, Crewe Business Park;Crewe May 16, 2017