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.

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Country Product Indication Sponsor Date
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes Khondrion BV August 10, 2015
CD33-directed antibody-drug conjugate consisting of an antibody conjugated to a DNA cross-linking pyrrolobenzodiazepine dimer drug Acute myeloid leukaemia Seattle Genetics UK, Limited August 10, 2015
Human allogeneic bone-marrow-derived osteoblastic cells Osteogenesis imperfecta Bone Therapeutics SA August 10, 2015
Glycyl-L-2-methylprolyl-L-glutamic acid Rett syndrome QRC Consultants Ltd August 10, 2015
Recombinant human acid ceramidase Cystic fibrosis Enzyvant Farber Ireland Limited August 10, 2015
Fixed-dose combination of fosfomycin disodium and tobramycin Cystic fibrosis CURx Pharma (UK) Limited August 10, 2015
Adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene Hunter Syndrome Laboratorios del Dr Esteve, S.A. August 10, 2015
Insulin human Short bowel syndrome Sirius Regulatory Consulting Limited August 10, 2015
Ibrutinib Marginal zone lymphoma Janssen-Cilag International NV August 10, 2015
Fibrinogen-coated albumin spheres Acute Radiation Syndrome Acute radiation syndrome Fibreu Limited August 10, 2015
Daratumumab Follicular lymphoma Janssen Research & Development, LLC;1400 McKean Road, P. O. Box 776;Spring House, Pennsylvania, 19477 August 6, 2015
Sodium valproate Wolfram syndrome The University of Birmingham;Birmingham Research Park - Vincent Drive, Birmingham B15 2SQ; August 5, 2015
Varlitinib Bile duct cancer ASLAN Pharmaceuticals;10A Bukit Pasoh Road, 089824; August 5, 2015
Butylidenephthalide Glioma Everfront Biotech, Inc.;11F., No. 31, Ln. 169, Kangning St., New Taipei City 221; August 5, 2015
Afatinib Non-small cell lung cancer Boehringer Ingelheim Pharmaceuticals, Inc.;900 Ridgebury Road, P. O. Box 368;Ridgefield, Connecticut, 06877 August 3, 2015
Inecalcitol Acute myeloid leukemia Hybrigenics, S.A.;3-5 Impasse Reille, 75014;Paris August 3, 2015
Doxorubicin Hepatoblastoma Double Bond Pharmaceutical AB July 28, 2015
Allogeneic human adult stem cells, isolated from skeletal muscle and expanded ex vivo Duchenne muscular dystrophy Karl Rouger July 28, 2015
Cannabidiol Perinatal asphyxia GW Pharma Ltd July 28, 2015
Humanised IgG4 monoclonal antibody against extracellular tau Progressive supranuclear palsy Biogen Idec Limited July 28, 2015
Triheptanoin Mitochondrial trifunctional protein deficiency Ultragenyx UK Limited July 28, 2015
Human plasminogen Plasminogen deficiency ProMetic BioTherapeutics Ltd July 28, 2015
Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate Amyotrophic lateral sclerosis PBS Regulatory Consulting Group Limited July 28, 2015
Synthetic double-stranded RNA oligonucleotide specific to hydroxyacid oxidase 1 gene Primary Hyperoxaluria Type 1 Dicerna EU Limited July 28, 2015
Synthetic hypericin Cutaneous T-cell lymphoma Kinesys Consulting Ltd July 28, 2015