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
Recombinant humanized immunoglobulin gamma 1 monoclonal antibody (mAb) directed against the CemX segment of human membrane-bound immunoglobulin E (mIgE) Hyper-IgE Syndrome Fountain Biopharma, Inc.;19F-1, No. 3, 3 Yuan Qu Street;Nangang District September 28, 2017
Tazemetostat Malignant pleural mesothelioma Epizyme Inc.;400 Technology Square, 4th Floor;Cambridge, Massachusetts, 02139 September 28, 2017
Adeno-associated viral vector serotype 5 encoding a microRNA targeted to human huntingtin gene Huntington disease uniQure Biopharma B.V.;113 Hartwell Avenue;Lexington, Massachusetts, 02421 September 27, 2017
Anti-STAT3 B VHH13 single domain antibody Pediatric osteosarcoma Singh Biotechnology, LLC;4708 Rue Bordeaux;Lutz, Florida, 33558 September 27, 2017
Fimaporfin Bile duct cancer PCI Biotech AS;64 Ullernchausseen;Ullern September 27, 2017
Rofecoxib Hemophilic arthropathy Tremeau Pharmaceuticals, Inc.;1 Broadway, 14th Floor;Cambridge, Massachusetts, 02142 September 26, 2017
Mitoxantrone hydrochloride liposome T-cell lymphoma CSPC Zhongqi Pharmaceutical Technology (Shijiazhuang) Co., Ltd.;., a subsidiary company within CSPC Pharmaceutical Group, Ltd, 226 Huanghe Avenue;Shijiazhuang Shi September 20, 2017
ICG136:CD123-CD33cCAR T-cells Acute myeloid leukemia iCell Gene Therapeutics;25 Health Sciences Drive, Suite 118;Stony Brook, New York, 11793 September 19, 2017
Non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNA Duchenne muscular dystrophy Nationwide Children's Hospital;Drug and Device Development Services, 700 Children's Drive;Columbus, Ohio, 43205 September 19, 2017
Prismocitrate 18 Use as replacement solution (includes regional anticoagulation of the extracorporeal circuit) in patients undergoing continuous renal replacement therapy (CRRT) Baxter Healthcare Corporation;32650 Wilson Road, WG1-3;Round Lake, Illinois, 60073 September 19, 2017
1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4-c]pyridin-6-yl)ethan-1-one Stargardt Disease Lin BioScience, LLC;3210 Merryfield Row;San Diego, California, 92121 September 19, 2017
Rimeporide Duchenne muscular dystrophy EspeRare Foundation;14, Ch des Aulx;Plan-les-Ouates September 19, 2017
Adeno-associated virus serotype 8 vector expressing B domain deleted codon optimized factor VIII Hemophilia A Baxalta US Inc.;1 Baxter Way;Westlake Village, California, 91362 September 18, 2017
Lixivaptan Polycystic kidney disease- type 1 Palladio Biosciences, Inc.;12 Penns Trail, Unit A;Newtown, Pennsylvania, 18940 September 18, 2017
Liposomal oxaliplatin Pancreatic adenocarcinoma KC Specialty Therapeutics, LLC;2002 West 39th Avenue;Kansas City, Arkansas, 66103 September 18, 2017
Nelatimotide and adegramotide (DSP-7888 dosing emulsion) Brain cancer Boston Biomedical, Inc.;640 Memorial Drive;Cambridge, Massachusetts, 02139 September 18, 2017
Tucatinib HER2+ colorectal cancer Cascadian Therapeutics, Inc.;2601 4th Avenue, Suite 500;Seattle, Washington, 98121 September 18, 2017
Priliximab Myelofibrosis Onco Therapies, Inc.;2265 East Foothill Boulevard;Pasadena, California, 91107 September 18, 2017
Recombinant human acid a-glucosidase co-administered with N-butyl-deoxynojirimycin (reconbinant human acid a-glucosidase/miglustat) Pompe disease Amicus Therapeutics, Inc.;1 Cedarbrook Drive;Cranbury Township, New Jersey, 08512 September 13, 2017
Rusalatide acetate Acute Radiation Syndrome Acute radiation syndrome Chrysalis BioTherapeutics, Inc.;2200 Market Street, Suite 609;Galveston, Texas, 77550 September 7, 2017
Dupilumab Eosinophilic esophagitis Eosinophilic esophagitis Regneron Pharmaceuticals, Inc.;777 Old Saw Mill River Road;Tarrytown, New York, 10591 September 5, 2017
N-(3-(2-(2,3-difluoro-4-(4-(2-hydroxyethyl)piperazin-1-yl)phenylamino)quinazolin-8-yl)phenyl)acrylamide Non-small cell lung cancer Checkpoint Therapeutics, Inc.;2 Gansevoort Street, 9th Floor;New York, New York, 10014 September 5, 2017
Elamipretide Primary mitochondrial myopathy Stealth BioTherapeutics;275 Grove Street, Suite 3-107;Newton, Massachusetts, 02466 September 5, 2017
Venetoclax Mantle cell lymphoma AbbVie Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 August 31, 2017
5-(4-carboxymethyl-phenylazo)-2-hydroxybenzoic acid disodium salt Ulcerative colitis Innovate Biopharmaceuticals, Inc.;8480 Honeycutt Road, Suite 200;Raleigh, North Carolina, 27615 August 31, 2017