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.

Become a Partner.

Country Product Indication Sponsor Date
Anti-sense oligonucleotide consisting of 2�0-Me RNA with a phosphorothioate backbone Cystic fibrosis ProQR Therapeutics III B.V.;Zernikedreef 9, 2333 CK Leiden, The Netherlands; September 23, 2013
Anti-sense oligonucleotide consisting of 2ÿƿÿ⿿ÿ⿿ÿ¢ÿƿÿ¢ÿ¢â⿿¬ÿ¡ÿ⿿ÿ¬ÿƿÿ¢ÿ¢â⿿¬ÿ¾ÿ⿿ÿ¢0-Me RNA with a phosphorothioate backbone Cystic fibrosis ProQR Therapeutics III B.V.;Zernikedreef 9, 2333 CK Leiden, The Netherlands; September 23, 2013
Brentuximab vedotin T-cell lymphoma Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 September 13, 2013
Anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A chain fusion protein Graft Versus Host Disease Xenikos BV;Molenveldlaan 152A, 6523RN;NIJMEGEN September 13, 2013
Autologous ex vivo expanded CD4+-enriched leukocytes treated with the de-methylating agent 5-aza-2â⿬⿢-deoxycytidine Glioblastoma CytoVac A/S;Science & Technology Park Scion DTU, DK-2970;Horsholm September 13, 2013
Autologous ex vivo expanded CD4+-enriched leukocytes treated with the de-methylating agent 5-aza-2â⿬⿢-deoxycytidine Glioblastoma multiforme CytoVac A/S;Science & Technology Park Scion DTU, DK-2970;Horsholm September 13, 2013
Autologous ex vivo expanded CD4+-enriched leukocytes treated with the de-methylating agent 5-aza-2’-deoxycytidine Glioblastoma multiforme CytoVac A/S;Science & Technology Park Scion DTU, DK-2970;Horsholm September 13, 2013
Aadeno-associated viral vector, serotype 2, containing the human choroideremia gene encoding human Rab escort protein 1 Choroideremia Sparks Therapeutics, Inc.;3737 Market Street, Suite 1300;Philadelphia, Pennsylvania, 19104 September 12, 2013
Evolocumab Homozygous Familial Hypercholesterolemia Amgen Inc.;One Amgen Center Drive, M/S 17 2 B;Thousand Oaks, California, 91320 September 12, 2013
L-arginyl-L-isoleucyl-L-valyl-L-prolyl-L-alanine-amide Acute Radiation Syndrome Acute radiation syndrome Soligenix, Inc.;29 Emmons Drive, Suite C-10;Princeton, New Jersey, 08540 September 11, 2013
Denosumab Hypercalcemia in malignancy Amgen, Inc.;One Amgen Center Drive, MS 17-2-C;Thousand Oaks, California, 91320 September 11, 2013
Dimethyl fumarate Friedreich Ataxia Ixchel Pharma, LLC;608 12th Street;Davis, California, 95616 September 11, 2013
Cysteamine Pancreatic Cancer Horizon Pharma USA, Inc.;150 S. Saunders Road;Lake Forest, Illinois, 60045 September 11, 2013
Nitric oxide Cystic fibrosis Novoteris, LLC;7245 Garden Grove Blvd., Suite C;Garden Grove, California, 92841 September 11, 2013
Dehydrated alcohol Hypertrophic obstructive cardiomyopathy Belcher Pharmaceuticals, LLC;6911 Bryan Dairy Road;Largo, Florida, 33777 September 11, 2013
Bendamustine hydrochloride with betadex sulfobutyl ether sodium Chronic lymphocytic leukemia Supratek Pharma, Inc.;6100 Royalmount Avenue, H4P 2R2;Quebec September 10, 2013
(4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane Chloride Progressive familial intrahepatic cholestasis Shire Human Genetic Therapies, Inc.;300 Shire Way;Lexington, MA, 02421-2101 September 4, 2013
Fenretinide T-cell lymphoma CerRx, Inc.;Reese Technology Center, 9801 Reese Blvd, Suite 104;Lubbock, Texas, 79416 September 4, 2013
(4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane Chloride Primary sclerosing cholangitis Shire Human Genetic Therapies, Inc.;300 Shire Way;Lexington, California, 02421 September 4, 2013
(4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane Chloride Alagille syndrome Shire Human Genetic Therapies, Inc.;300 Shire Way;Lexington, Massachusetts, 02421 September 4, 2013
(4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane Chloride Primary biliary cirrhosis Shire Human Genetic Therapies, Inc.;300 Shire Way;Lexington, Massachusetts, 02421 September 4, 2013
Maralixibat Cholestasis Shire Human Genetic Therapies, Inc.;300 Shire Way;Lexington, Massachusetts, 02421 September 4, 2013
Angiotensin (1-7) Patients requiring stem cell transplantation to accelerate the mobilization of hematopoietic stem cells (CD34+) from the bone marrow to the peripheral blood when combined with a granulocyte colony-stimulating factor US Biotest, Inc.;231 Bonetti Dr.,, Suite 240;San Luis Obispo, California, 93401 August 30, 2013
Expanded human allogeneic neural retinal progenitor cells extracted from neural retina Retinitis pigmentosa ReNeuron Ltd;Pencod Business Park, Pencod, Bridgend; August 22, 2013
Deflazacort Duchenne muscular dystrophy PTC Therapeutics, Inc.;100 Corporate Court;South Plainfield, New Jersey, 07080 August 16, 2013