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
Patidegib Nevoid basal cell carcinoma syndrome PellePharm, Inc.;275 Middlefield Road;Menlo Park, California, 94025 July 19, 2017
Filgotinib maleate pediatric Crohn’s disease Gilead Sciences, Inc.;199 E. Blaine St.;Seattle, Washington, 98102 July 18, 2017
One, two, three, or four antisense oligonucleotides of Phosphorodiamidate morpholino oligomer combination that skips exons 4, 5, 6, and 7 of the gamma sarcoglycan (SGCG) gene Limb-girdle muscular dystrophy Kurt+Peter Foundation;P. O. Box 691;Menlo Park, California, 94025 July 18, 2017
Ustekinumab Pediatric systemic lupus erythematosus Janssen Biotech, Inc.;800 Ridgeview Drive;Horsham, Pennsylvania, 19044 July 18, 2017
Tirapazamine Hepatocellular Carcinoma PhaRA bvba July 17, 2017
(S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine Gastrointestinal stromal tumours PhaRA bvba July 17, 2017
Oxymetazoline hydrochloride Spinal cord injury RDD Pharma Limited July 17, 2017
Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor Diffuse Large B-cell Lymphoma Celgene Europe Limited July 17, 2017
Bacillus subtilisoxalate decarboxylase Primary hyperoxaluria Allena Pharmaceuticals Ireland Limited July 17, 2017
Recombinant human antibody directed against misfolded human superoxide dismutase 1 Amyotrophic lateral sclerosis The Medical & Regulatory Partnership Limited July 17, 2017
Polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(1?3)-beta-D-galactopyranoside Anti-MAG neuropathy SFL Regulatory Affairs Consulting Ltd July 17, 2017
Retinol Retinopathy of prematurity orphanix GmbH July 17, 2017
Sirolimus Pachyonychia Congenita Raremoon Consulting Ltd July 17, 2017
Ranpirnase Ebola Tamir Biotechnology, Inc.;51 John F Kennedy Parkway, 1st Floor West;Short Hills, New Jersey, 07078 July 14, 2017
(20S)-7-tert-butyldimethysilyl-10-hydroxycamptothecin Glioblastoma Vivacitas Oncology, Inc.;1990 North California Boulevard, 8th Floor;Walnut Creek, California, 94596 July 13, 2017
Autologous mononuclear cells derived from umbilical cord blood Severe pediatric congenital heart disease Timothy Nelson, MD, PhD;Mayo Clinic, Hypoplastic Left Heart Syndrome Program;Rochester, Minnesota, 55902 July 13, 2017
Electrokinetically altered saline solution with an elevated dissolved oxygen concentration Amyotrophic lateral sclerosis Revalesio Corporation;1202 East D Street;Tacoma, Washington, 98421 July 10, 2017
Cosyntropin Duchenne muscular dystrophy Mallinckrodt ARD Inc.;675 McDonnell Boulevard;Hazelwood, Missouri, 63042 July 6, 2017
Blinatumomab Diffuse Large B-cell Lymphoma Amgen Inc.;1 Amgen Center Drive;Thousand Oaks, California, 91320 July 6, 2017
Humanized immunoglobulin G4-variant monoclonal antibody directed against the p19 subunit of interleukin (IL)-23 pediatric Crohn’s disease Eli Lilly & Company;Lilly Corporate Center;Indianapolis, Indiana, 46285 July 6, 2017
Entrectinib NTRK fusion-positive solid tumors Ignyta, Inc.;4545 Towne Centre Court;San Diego, California, 92121 July 5, 2017
Tadekinig alfa Still’s disease AB2 Bio Ltd;EPFL Innovation Park, Building B;Lausanne July 3, 2017
Sirukumab Juvenile idiopathic arthritis (JIA) in pediatric patients Janssen Research & Development, LLC;1400 McKean Road, P.O. Box 776;Spring House, Pennsylvania, 19477 July 3, 2017
Small Molecule Bruton’s Agammaglobulinemia Tyrosine Kinase (BKT) Inhibitor Pemphigus vulgaris Principia Biopharma Inc.;400 East Jamie Court;South San Francisco, California, 94080 June 29, 2017
Retinol palmitate (vitamin A palmitate) Retinopathy of prematurity orphanix GmbH;41 Peter-Rosegger-Stra�e;Ried im Innkreis June 28, 2017