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
Solnatide Generalized systemic pseudohypoaldosteronism type 1 (also known as autosomal recessive PHA-I or PHA-IB) Apeptico Forschung und Entwicklung GmbH;Mariahilferstrabe 136, Top 1150;Vienna February 8, 2016
4-[(2E)-1-Oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide Retinitis pigmentosa Shire HGT, Inc.;300 Shire Way;Lexington, Massachusetts, 02421 February 8, 2016
Echinocandin B,1-[(4R,5R)-4-hydroxy-N2-[[4��-(pentyloxy)[1,1�:4�,1��-terphenyl]-4-yl]carbonyl]-5-[2-(trimethylammonio)ethoxy]-L-ornithine]-4-[(4S)-4-hydroxy-4-(4-hydroxyphenyl)-L-allothreonine] Candidemia and invasive candidiasis infections caused by susceptible Candida spp Cidara Therapeutics, Inc.;6310 Nancy Ridge Drive, Suite 101;San Diego, California, 92121 February 8, 2016
Eltoprazine HCl Levodopa-induced dyskinesia associated with Parkinson's disease Amarantus BioScience Holdings, Inc.;655 Montgomery St., Suite 900;San Francisco, California, 94111 February 8, 2016
Midazolam Seizures induced by organophosporous insecticide poisoning Meridian Medical Technologies, Inc.;A Pfizer Company, 6350 Stevens Forest Road;Columbia, Maryland, 21046 February 8, 2016
Rezafungin Candidemia and invasive candidiasis infections caused by susceptible Candida spp Cidara Therapeutics, Inc.;6310 Nancy Ridge Drive, Suite 101;San Diego, California, 92121 February 8, 2016
Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 (HAO1) mRNA Primary Hyperoxaluria Type 1 Alnylam Pharmaceuticals, Inc.;300 Third Street;Cambridge, Massachusetts, 02142 February 8, 2016
Angiotensin (1-7) LAMA2-related muscular dystrophy Tarix Orphan, LLC;12 Bow Street;Cambridge, Massachusetts, 02138 February 8, 2016
Evinacumab Treat of homozygous familial hypercholesterolemia Regeneron Pharmaceuticals, Inc.;777 Old Saw Mill River Road;Tarrytown, New York, 10591 February 8, 2016
Human recombinant dual-variable domain immunoglobulin G1 monoclonal antibody that selectively neutralizes tumor necrosis factor-alpha and interkeukin-17A Pediatric patients 0 to <18 yrs of age with oligoarticular-course and polyarticular-course juvenile idiopathic arthritis AbbVie, Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 February 4, 2016
Venetoclax Acute myeloid leukemia AbbVie Inc.;1 Waukegan Road, Dept PA77, Bldg. AP30;North Chicago, Illinois, 60044 February 4, 2016
Tazemetostat Malignant rhabdoid tumors (MRTs) Epizyme Inc.;400 Technology Square, 4th Floor;Cambridge, Massachusetts, 02139 February 4, 2016
Midazolam Status epilepticus Meridian Medical Technologies, Inc.;A Pfizer Company, 6350 Stevens Forest Road;Columbia, Maryland, 21046 February 4, 2016
Afamelanotide Cutaneous variants of porphyria (which includes treatment and prevention of cutaneous manifestations of disease) Clinuvel Inc.;40 Worth Street, Suite 1053;New York, New York, 10013 February 4, 2016
Somatic cell therapy product containing hematopoietic progenitor cells (HPC), Facilitating Cells (FC), and alpha beta T cells. Prevention of organ rejection in living donor kidney transplant recipients Regenerex LLC;570 South Preston Street, Suite 404;Louisville, Kentucky, 40202 February 4, 2016
Synthesized peptide of the L-amino acids with the sequence H-Lys-Glu-Phe-Leu-His-Pro-Ser-Lys-Val-Asp-Leu-Pro-Arg-OH Ovarian cancer Soricimed Biopharma Inc.;18 Botsford Street, Suite 201;Moncton, New Brunswick February 4, 2016
Allogeneic Epstein-Barr virus cytotoxic T lymphocytes Epstein-Barr virus-positive post-transplant lymphoproliferative disorders ATARA Biotherapeutics, Inc.;4360 Park Terrace Drive, Suite 100;Westlake Village, California, 91361 February 4, 2016
Solnatide Primary graft dysfunction following lung transplantation Apeptico Forschung und Entwicklung GmbH;c/o mingo bueros, 1150;Vienna February 4, 2016
Ibrutinib Patients with extranodal marginal zone lymphoma (mucosa associated lymphoid tissue [MALT type] lymphoma) Pharmacyclics, LLC;995 E. Arques Avenue;Sunnyvale, California, 94085 February 2, 2016
Oligodendrocyte progenitor cells Acute spinal cord injury Asterias Biotherapeutics;6300 Dumbarton Circle;Fremont, California, 94555 February 2, 2016
Bevacizumab Malignant pleural mesothelioma Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 January 27, 2016
Endothelin receptor B antagonist Malignant melanoma Stages IIB to IV ENB Therapeutics, LLC;166 5th Avenue, 2nd Floor;New York, New York, 10010 January 21, 2016
HLA-A2-selective gp100-specific T cell redirector Uveal melanoma Immunocore Ltd;Milton Park, 90 Park Drive;Milton January 21, 2016
Small molecule inhibitor of Exon 17 Mutant KIT Mastocytosis Blueprint Medicines Corporation;38 Sidney Street, Suite 200;Cambridge, Massachusetts, 02139 January 21, 2016
Human/Murine Chimeric Monoclonal antibody to Endoglin Soft tissue sarcoma Tracon Pharmaceuticals, Inc.;4350 LaJolla Village Dr., Suite 800;San Diego, California, 92122 January 20, 2016