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
Triclabendazole Fascioliasis Tropicalis Pharmaceuticals, LLC;22345 Bracketts Road;Excelsior, Minnesota, 55331 June 27, 2016
Molgramostim Acute respiratory distress syndrome Savara ApS June 27, 2016
Cyclocreatine Creatine deficiency syndromes Pharma Gateway AB June 27, 2016
Recombinant humanised monoclonal IgG2 lambda antibody against human sclerostin Osteogenesis imperfecta Mereo Biopharma 3 Limited June 27, 2016
Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment Treatment in haematopoietic stem cell transplantation Kiadis Pharma Netherlands B.V. June 27, 2016
Teriparatide Hypoparathyroidism Alacrita LLP June 27, 2016
Humanised anti-IL-6 receptor monoclonal antibody Neuromyelitis optica Chugai Pharma Europe Ltd June 27, 2016
(S)-7-(1-acryloylpiperidin-4-yl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydroprazolo[1,5-a]pyrimidine-3-carboxamide Mantle cell lymphoma BeiGene USA, Inc.;2115 Linwood Avenue, 4th Floor;Fort Lee, New Jersey, 07024 June 23, 2016
Ibrutinib Chronic Graft versus Host disease Pharmacyclics, LLC;995 East Arques Avenue;Sunnyvale, California, 94085 June 23, 2016
7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one*2HCl Glioblastoma Oncoceutics, Inc.;3624 Market Street, Suite 5E;Philadelphia, Pennsylvania, 19104 June 23, 2016
Sperm Protein-17 oral microparticle vaccine Ovarian cancer Kiromic, LLC;2707 Fannin Street, Fannin St. Professional Building;Houston, Texas, 77002 June 21, 2016
Sirolimus Beta-thalassemia Rare Partners srl Impresa Sociale;31 Corso Magenta;Milano June 21, 2016
Varlitinib Stomach Cancer ASLAN Pharmaceuticals;10A Bukit Pasoh Road; June 20, 2016
Napabucasin Stomach Cancer Boston Biomedical, Inc.;640 Memorial Drive;Cambridge, Massachusetts, 02139 June 20, 2016
Tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride West syndrome Anavex Life Sciences Corp.;51 West 52nd Street, 7th Floor;New York, New York, 10019 June 20, 2016
Hydromorphone hydrochloride (intrathecal infusion) Reflex sympathetic dystrophy syndrome Piramal Critical Care, Inc.;261 Brodhead Road, Suite 221;Bethlehem, Pennsylvania, 18017 June 16, 2016
Antisense oligonucleotide targeting the U isoform of SNP rs362307 Huntington disease Wave LIfe Sciences;733 Concord Avenue;Cambridge, Massachusetts, 02138 June 16, 2016
L-pyr-L-glu-L-gln-L-leu-L-glu-L-arg-L-ala-L-leu-L-asn-L-ser-L-ser Sarcoidosis Araim Pharmaceuticals, Inc.;580 White Plains Road, Suite 210;Tarrytown, New York, 10591 June 13, 2016
L-pyr-L-glu-L-gln-L-leu-L-glu-L-arg-L-ala-L-leu-L-asn-L-ser-L-ser Treatment to increase survival and improve functioning of pancreatic islets following transplantation Araim Pharmaceuticals, Inc.;580 White Plains Road, Suite 210;Tarrytown, New York, 10591 June 13, 2016
Fosbretabulin tromethamine Glioma Mateon Therapeutics, Inc.;701 Gateway Boulevard, Suite 210;South San Francisco, California, 94080 June 8, 2016
Recombinant, adeno-associated virus serotype 2 vector carrying linear single-stranded AADC mRNA cDNA Aromatic amino acid decarboxylase deficiency Agilis Biotherapeutics, LLC;Kendall Square, 245 First Street;Cambridge, Massachusetts, 02142 June 8, 2016
Recombinant human cerebral dopamine neurotrophic factor Amyotrophic lateral sclerosis Herantis Pharma Plc;4 Viikinkaari;Helsinki June 8, 2016
Glibentek (glibenclamide oral suspension) Neonatal diabetes (monogenic forms of diabetes that present in infancy) AMMTeK;15 Rue B�ranger;Paris June 8, 2016
Folate binding protein peptide: Glu-Ile-Trp-Thr-His-Ser-Tyr-Lys-Val Ovarian cancer Galena Biopharma, Inc.;Crow Canyon Place, Suite 380;San Ramon, California, 94583 June 8, 2016
Folate Binding Protein Peptide: Glu-Ile-Trp-Thr-Phe-Ser-Thr-Lys-Val Ovarian cancer Galena Biopharma, Inc.;Crow Canyon Place, Suite 380;San Ramon, California, 94583 June 8, 2016