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
Adeno-associated viral vector expressing the human vascular endothelial growth factor gene Critical limb ischemia in no-option patients where other procedures, grafts, or angioplasty are not indicated Integene International Holding, LLC;1951 NW 7th Avenue, Suite 300;Miami, Florida, 33136 December 31, 2015
Gemcitabine Upper tract urothelial carcinoma TARIS Biomedical LLC;113 Hartwell Avenue;Lexington, Massachusetts, 02421 December 30, 2015
Pembrolizumab Hodgkin’s disease Merck Sharp & Dohme Corp. a subsidiary of Merck & Co., Inc;126 E. Lincoln Avenue, P. O. Box 2000;Rathway, Pennsylvania, 07065 December 30, 2015
Allogeneic ex-vivo expanded placental adherent stromal cells For the treatment of severe preeclampsia Pluristem Therapeutics, Inc;MATAM Advanced Technology Park, Building # 5;Haifa December 29, 2015
Fosbretabulin tromethamine Neuroendocrine tumors Mateon Therapeutics, Inc.;701 Gateway Blvd, Suite 210;South San Francisco, California, 94080 December 29, 2015
Antisense oligonucleotide targeted to the huntingtin protein Huntington disease Isis Pharmaceuticals, Inc.;2855 Gazelle Court;Carlsbad, California, 92010 December 29, 2015
Noscapine Amyotrophic lateral sclerosis KineMed Inc.;5980 Horton Street, Suite 470;Emeryville, California, 94608 December 29, 2015
Dipraglurant Levodopa-induced dyskinesias Addex Pharma SA;Chemin des Aulx 14, 1228 Plan-Les-Ouates;Geneva December 29, 2015
Ozanimod Ulcerative colitis Celgene Corporation;3033 Science Park Road, Suite 300;San Diego, California, 92121 December 29, 2015
Cyclo{-Asn-Leu-d-Phe-Al[(N5-acetyl-N5-hydroxy-Orn)-(N5-acetyl-N5-hydroxy-Orn)-(N5-hydroxy-Orn)]-} . Al(III) Invasive aspergillosis Vical Incorporated;10390 Pacific Center Court;San Diego, California, 92121 December 29, 2015
Recombinant adeno-associated virus serotype AAV8 vector encoding human ornithine transcarbamylase Ornithine Transcarbamylase Deficiency Dimension Therapeutics;840 Memorial Drive;Cambridge, Massachusetts, 02139 December 29, 2015
Treprostinil sodium solution enclosed within a pre-filled, pre-programmed, single-use, electronically-controlled infusion pump Pulmonary Arterial Hypertension SteadyMed Therapeutics;2603 Camino Ramon, Suite 350;San Ramon, California, 94583 December 29, 2015
Fluticasone propionate Eosinophilic esophagitis Eosinophilic esophagitis Banner Life Sciences, LLC;4125 Premier Drive;High Point, North Carolina, 27265 December 29, 2015
2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl) methoxy)benzaldehyde Sickle cell anemia Global Blood Therapeutics, Inc.;400 East Jamie Court, Suite 101;South San Francisco, California, 94080 December 29, 2015
Human Wilms tumor protein 1 peptides Acute myeloid leukemia SELLAS Life Sciences Group;75 Baarerstrasse;Zug December 23, 2015
Imetelstat Myelodysplastic syndrome Janssen Research & Development, LLC;920 U.S. 202;Raritan, New Jersey, 08869 December 23, 2015
Magnesium lysinate bis eicosapentaenoate Familial adenomatous polyposis Thetis Pharmaceuticals;167 Old Post Road, 2nd Floor;Southport, Connecticut, 06890 December 23, 2015
Rovalpituzumab tesirine Small cell lung cancer AbbVie Inc.;Dept. PA77, Bldg. AP30-1, 1 Waukegan Road;Lake Bluff, Illinois, 60044 December 22, 2015
Substituted imidazopyridine amide Active tuberculosis (TB) Qurient Co., Ltd.;C-dong 8th floor, 242 Pangyo-ro, Bundang-gu, Seongnam-si;Gyeonggi-do December 22, 2015
Carbon dioxide Trigeminal neuralgia Capnia, Inc.;3 Twin Dolphin Drive, Suite 160;Redwood City, California, 94065 December 17, 2015
Stemchymal Polyqlutamine spinocerebellar ataxia Steminent Biotherapeutics, Inc.;No. 16 Wenhu Street, Neihu Dist.; December 16, 2015
Live attenuatedListeria monocytogenesdeltaactA/deltainlBstrain expressing human mesothelin Malignant pleural mesothelioma Aduro Biotech Holdings, Europe B.V. December 14, 2015
Bilayer engineered collagen hydrogel-based skin graft composed of autologous keratinocytes and fibroblasts Partial deep dermal and full thickness burns Voisin Consulting S.A.R.L. December 14, 2015
Sodium (2R,3S,5R)-5-(4-amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5-(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetrahydrofuran-2-yl)methyl phosphate Acute myeloid leukaemia Otsuka Pharmaceutical Europe Ltd. December 14, 2015
Variant of recombinant human fibroblast growth factor 19 Primary sclerosing cholangitis Diamond BioPharm Limited December 14, 2015