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
An adeno-associated viral vector containing a codon-optimised human factor IX gene (AAV5-hFIXco) Hemophilia B uniQure, B.V.;Meiberdreef 61, 1100 DA;Amsterdam December 22, 2011
Delta-1,4,9(11)-pregnatriene-17-alpha,21-dihydroxy-16-alpha-methyl-3,20-dione Duchenne muscular dystrophy ReveraGen Biopharma;9700 Great Seneca Highway;Rockville, MD, 20850 December 2, 2011
Vamorolone Duchenne muscular dystrophy ReveraGen Biopharma;155 Gibbs Road, Suite 433;Rockville, Maryland, 20850 December 2, 2011
Delta-1,4,9(11)-pregnatriene-17-alpha,21-dihydroxy-16-alpha-methyl-3,20-dione Duchenne muscular dystrophy ReveraGen Biopharma;9700 Great Seneca Highway;Rockville, MD, 20850 December 2, 2011
5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzp[d]oxazole Duchenne muscular dystrophy Summit Corporation plc;91 Milton Park, Oxfordshire OX14 4RY; November 22, 2011
MCMV5322A/MCMV3068A Cytomegalovirus retinitis Genentech;1 DNA Way, MS#241B;South San Francisco, California, 94080 November 22, 2011
Autologous lymphoma-derived immunoglobulin idiotype antigen vaccine conjugated to keyhole limpet hemocyanin Waldenstrom macroglobulinemia Biovest International, Inc.;324 S. Hyde Park Avenue, Suite 350;Tampa, Florida, 33606 October 18, 2011
Eculizumab Shiga-Toxin producing escherichia coli hemolytic uremic syndrome Alexion Pharmaceuticals, Inc.;352 Knotter Drive;Cheshire, Connecticut, 06410 October 18, 2011
(-)-(3aR,4S,7aR)-4-Hydroxy-4-m-tolylethynyl-octahydro-indole-1-carboxylic acid methyl ester Fragile X syndrome Novartis Pharmaceuticals Corp.;One Health Plaza;East Hanover, New Jersey, 07936 October 12, 2011
Abeotaxane inhibitor of microtubules Gliomas Cortice Biosciences;1345 Avenue of the Americas, 42nd Floor;New York, New York, 10105 October 7, 2011
Ferumoxytol For use in magnetic resonance imaging in brain metastases Oregon Health & Science University;3181 SW Sam Jackson Pk Road, Mailcode L603;Portland, Oregon, 97239 October 7, 2011
4-[2-(3-Propyl-[1,2,4]0xadiazol-5-yl)-vinyl]-benzene-1,2-diol Chronic myeloid leukemia Piramal Enterprises Limited;1 Nirlon Complex, Goregaon (East) 400063, Maharashtra;Mumbai October 4, 2011
Aceneuramic acid Hereditary inclusion body myopathy Ultragenyx Pharmaceutical, Inc.;60 Leveroni Court, Suite 200;Novato, California, 94949 September 23, 2011
4-[[9-(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol Idiopathic pulmonary fibrosis Celgene Corporation;33 Technology Drive;Warren, New Jersey, 07059 September 23, 2011
Dendritic hybrid cell vaccine Neuroblastoma Orbis Health Solutions, LLC;111 Smith Hines Road, Suite E & F;Greenville, South Carolina, 29607 September 23, 2011
Bispecific antibody targeting interleukin 4 (IL-4) and interleukin 13 (IL-13) Idiopathic pulmonary fibrosis Sanofi-Aventis US, Inc.;200 Crossing Blvd;Bridgewater, New Jersey, 08807 September 14, 2011
Cardiotrophin-1 Acute liver failure Digna Biotech S.L.;C/Boix y Morer 6-8, 28003 Madrid; September 13, 2011
L-pyr-L-glu-L-gln-L-leu-L-glu-L-arg-L-ala-L-leu-L-asn-L-ser-L-ser Sarcoidosis Araim Pharmaceuticals, Inc.;712 Kitchawan Road;Ossining, New York, 10562 September 13, 2011
Angiotensin-(1-7) Pulmonary arterial hypertension US Biotest, Inc.;231 Bonetti Drive, Suite 240;San Luis Obispo, California, 93401 September 13, 2011
Lanreotide acetate Carcinoid syndrome Ipsen Biopharmaceuticals, Inc.;106 Allen Road;Basking Ridge, New Jersey, 07920 September 8, 2011
Domperidone Hypoprolactinemia in breastfeeding mothers, and in some hypoprolactinemic conditions following the use of cabergoline or bromocriptine in mothers who wish to initiate or return to breastfeeding Thomas W. Hale, RPh, PhD;Texas Tech University Health Sciences Ctr., 1400 Coulter Suite 1400;Amarillo, Texas, 79106 September 2, 2011
Deferiprone Siderosis ApoPharma, Inc.;200 Barmac Drive; September 1, 2011
Elotuzumab Multiple myeloma Bristol-Myers Squibb Co.;Route 206 & Province Line Road;Princeton, New Jersey, 08540 September 1, 2011
HPV16 E6/E7 synthetic long peptides vaccine Epithelial neoplasias of the vulva positive for human papilloma virus type 16 ISA Therapeutics BV;J.H. Oortweg 19, 2333 CH Leiden; September 1, 2011
1-(3-chloro-5-{[4-(4-chloro-2-thienyl)-5-(4-cyclohexylpiperazin-l-yl)-1,3-thiazol-2-yl]carbamoyl}- 2-pyridyl) piperidine-4-caboxylic acid Monomaleate Idiopathic thrombocytopenic purpura Dova Pharmaceuticals, Inc.;240 Leigh Farm Road, Suite 245;Durham, North Carolina, 27707 September 1, 2011