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
Oxfendazole Cysticercosis Oxfendazole Development Group;736 Cathcart Road;Blue Bell, Pennsylvania, 19422 August 7, 2017
KIT and PDGFR inhibitor Glioblastoma Deciphera Pharmaceuticals, LLC;500 Totten Pond Road;Waltham, Massachusetts, 02451 August 7, 2017
Odiparcil Mucopolysaccharidosis type VI Inventiva Pharma;50 rue de Dijon, Daix 21121; August 3, 2017
Recombinant B-domain deleted human coagulation factor VIII Fc-von Willebrand factor-XTEN fusion protein Hemophilia A Bioverativ Therapeutics, Inc.;225 2nd Avenue;Waltham, Massachusetts, 02451 August 3, 2017
Seletalisib Activated phosphoinositide 3-kinase delta syndrome (APDS) UCB, Inc.;1650 Lake Park Drive;Smyrna, Georgia, 30080 August 3, 2017
Tesevatinib Non-small cell lung cancer Kadmon Corporation, LLC;450 East 29th Street;New York, New York, 10016 August 3, 2017
Granexin� gel (aCTI peptide) Cutaneous radiation injury due to radiation disaster FirstString Research, Inc.;300 West Coleman Boulevard, Suite 203;Mount Pleasant, South Carolina, 29464 August 3, 2017
Papaverine Mesenteric ischemia Nir Barak, MD;CO Noa Avtalion, 161 Jefferson Avenue;Tenafly, New Jersey, 07670 August 3, 2017
Human monoclonal antibody inhibitor of mannan-binding lectin-associated serine protease-2 IgA Nephropathy Omeros Corporation;201 Elliott Avenue West;Seattle, Washington, 98119 August 3, 2017
Miltefosine Schistosomiasis Profounda, Inc.;5790 Hoffner Avenue, Suite 507;Orlando, Florida, 32822 August 2, 2017
Gemfibrozil Neuronal ceroid lipofuscinoses Polaryx Therapeutics, Inc.;140 East Ridgewood Avenue, Suite 415, South Tower;Paramus, New Jersey, 07652 August 2, 2017
1-{2-[3-(3,4-Dichlorophenyl)ureido]-6-methyl-pyrimidin-4-yl}-amino-3(dimethylamino)propane Di methansulfonate Glioma Curtana Pharmaceuticals, Inc.;1624 Headway Circle;Austin, Texas, 78754 August 2, 2017
Carisbamate Lennox-Gastaut syndrome SK Life Sciences, Inc.;22-10 New Jersey 208 South;Fair Lawn, New Jersey, 07410 August 2, 2017
Cosyntropin West syndrome West Therapeutic Development, LLC;540 Ziegler Drive;Grayslake, Illinois, 60030 August 2, 2017
Crofelemer Short bowel syndrome Napo Pharmaceuticals, Inc.;201 Mission Street, Suite 2375;San Francisco, California, 94105 August 2, 2017
Cipargamin Malaria Paul Maher, MD;10644 Weymouth Street, Suite 104;Bethesda, Maryland, 20814 July 27, 2017
Humanized immunoglobulin G1 (IgG1) T-cell bispecific (TCB) antibody targeting carcinoembryonic antigen (CEA) and CD3 epsilon chain (CD3e) on T-cells Pancreatic Cancer Genentech, Inc.;1 DNA Way, MS 35-5E;South San Francisco, California, 94080 July 27, 2017
Picropodophyllin Glioma Axelar AB;Karolinska Institutet Science Park, 2 Fogdevreten;Solna July 27, 2017
Pro-Pro-Thr-Val-Pro-Thr-Arg Xeroderma pigmentosum ProGeLife S.A.S;8 Rue Sainte-Barbe;Marseille July 27, 2017
Combination of sodium phenylbutyrate and tauroursodeoxycholic acid dihydrate Amyotrophic lateral sclerosis Amylyx Pharmaceuticals Inc.;210 Broadway, Suite 201;Cambridge, Massachusetts, 02139 July 27, 2017
Cis-diamminedichloroplatinum (II) polyethylene glycol-polyglutamate (cis platinum PEG-pGlu) polymeric micelles Biliary tract carcinoma NanoCarrier Co., Ltd.;144-15 Chuno Gaiku, 226-39 Wakashiba;Kashiwa July 27, 2017
Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol palmitoylated linker Hemoglobinuria Ra Pharmaceuticals, Inc.;87 Cambridge Park Drive;Cambridge, Massachusetts, 02140 July 19, 2017
(R)-3-[3-Fluoro-4-(1-methyl-1,4,5,6-tetrahydro-1,2,4-triazin-4-yl)phenyl]-5(R)-(hydroxymethyl)oxazolidin-2-one Tuberculosis LegoChem Biosciences, Inc.;8-26 Munpyeongseo-ro;Daejeon July 19, 2017
Artemisone Malaria Artemis Therapeutics, Inc.;18 East 16th Street, Suite 307;New York, New York, 10003 July 19, 2017
Fusion protein linking human frataxin to a cell-penetrant peptide Friedreich Ataxia Chondrial Therapeutics, Inc.;150 Monument Road;Bala Cynwyd, Pennsylvania, 19004 July 19, 2017