Treatments

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

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

Become a Partner.

Country Product Indication Sponsor Date
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
Palm tocotrienol complex CADASIL Hovid Berhad;121 Jalan Tunku Abdul Rahman;Ipoh June 13, 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
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
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
Resiquimod Cutaneous T-cell lymphoma Galderma Research and Development, LLC;14501 North Freeway;Fort Worth, Texas, 76177 May 24, 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
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