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
Immune globulin infusion 10% (human) with recombinant human hyaluronidase) Chronic inflammatory demyelinating polyneuropathy Baxalta US, Inc.;1 Baxter Way;Westlake Village, California, 91362 March 31, 2016
Gentamicin C2 congener Cystic fibrosis La Jolla Pharmaceuticals Company;10182 Telesis Court, Suite 600;San Diego, California, 92121 March 29, 2016
3-(5-Fluoro-benzofuran-3-yl)-4-(5-methyl-5H-[1,3]dioxolo[4,5-f]indol-7-yl)-pyrrole-2,5-dione Glioblastoma Actuate Therapeutics;1401 Foch Street, Suite 140;Fort Worth, Texas, 76107 March 29, 2016
Autologous CD4+/CD8+ T cells transduced with lentiviral vector directed against the NY-ESO-1 protein Soft tissue sarcoma Adaptimmune, LLC;Two Commerce Square, 2001 Market Street;Philadelphia, Pennsylvania, 19103 March 28, 2016
131I-BC8 monoclonal antibody (Anti-CD45 murine monoclonal antibody radiolabeled with 131I Acute myeloid leukemia Actinium Pharmaceuticals, Inc.;379 Thornall Avenue, 6th Floor;Edison, New Jersey, 08837 March 28, 2016
Ivabradine Dilated cardiomyopathy Amgen, Inc.,;1 Amgen Center Drive;Thousand Oaks, California, 91320 March 23, 2016
Lentiviral vector containing the human liver and erythroid pyruvate kinase gene Pyruvate kinase deficiency Consorcio Centro de Investigaci�n Biom�dica en Red, M.P.;Monforte de Lemos, 3-5 Pabellion 11;Madrid March 23, 2016
Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues Primary hyperoxaluria Alnylam UK Limited March 21, 2016
Ubenimex Pulmonary Arterial Hypertension Eiger Biopharmaceuticals Europe Limited March 21, 2016
Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene Haemophilia A BioMarin Europe Ltd March 21, 2016
Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase Ornithine Transcarbamylase Deficiency Pharma Gateway AB March 21, 2016
Acalabrutinib small lymphocytic lymphoma Acerta Pharma, BV March 21, 2016
Acalabrutinib Mantle cell lymphoma Acerta Pharma, BV March 21, 2016
Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes Post-transplant lymphoproliferative disorder Atara Biotherapeutics Ireland Limited March 21, 2016
Acalabrutinib Waldenstrom macroglobulinemia Acerta Pharma, BV March 21, 2016
Diaspirin cross-linked haemoglobin Oesophageal cancer New B Innovation (UK) Limited March 21, 2016
Exenatide Idiopathic intracranial hypertension Alan Boyd Consultants Ltd March 21, 2016
Fenretinide Cutaneous T-cell lymphoma Clinipace GmbH March 21, 2016
Glucopyranosyl lipid A stable emulsion and recombinant New York esophageal squamous cell carcinoma-1 protein Soft tissue sarcoma Immune Design Ltd March 21, 2016
Florilglutamic acid (18F) Glioma Piramal Imaging GmbH March 21, 2016
Sindbis virus envelope pseudotyped lentiviral vector encoding New York esophageal squamous cell carcinoma-1 protein Soft tissue sarcoma Immune Design Ltd March 21, 2016
Florilglutamic acid (18F) Hepatocellular Carcinoma Piramal Imaging GmbH March 21, 2016
Fosbretabulin tromethamine Gastro-entero-pancreatic neuroendocrine tumours Diamond BioPharm Limited March 21, 2016
N-acetyl-D-mannosamine monohydrate GNE myopathy Escala Therapeutics Ltd March 21, 2016
Guanfacine and amphetamine Tourette syndrome Genco Sciences, LLC;1011 Greenwood Avenue;Willmette, Illinois, 60091 March 17, 2016