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
Autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human BA-T87Q-globin gene Sickle cell anemia bluebird bio Inc.;840 Memorial Dr., 4th Floor;Cambridge, Massachusetts, 02139 February 26, 2014
(6-[4-Deoxy-4-[(2E,4E)-tetradecadienoylglycyl] amino-L-glyceroB-L-manno-heptopyranosyl]amino-9H-purine) Parenteral treatment of painful, chronic, chemotherapy-induced peripheral neuropathy that is refractory to conventional analgesics DARA Therapeutics, Inc.;8601 Six Forks Road, Suite 160 (Forum 1);Raleigh, North Carolina, 27615 February 21, 2014
Doxofylline Bronchiectasis Alitair Pharmaceuticals, Inc.;55 Madison Avenue, Suite 400;Morristown, New Jersey, 07960 February 14, 2014
Bisantrene HCl Acute myeloid leukemia Race Oncology, Ltd;50 Kings Park Road, Level 2;West Perth WA 6005 February 14, 2014
Caprine hyperimmune serum against HIV lysate Systemic scleroderma Daval International Limited;4a Gildredge Road,, East Sussex, BN21 4RL;East Sussex February 14, 2014
REMUNE HIV 1 Pediatric HIV/AIDS (age through 16 years) Immune Response BioPharma, Inc.;1801 Atlantic Avenue, Suite 8352;Atlantic City, New Jersey, 08401 February 14, 2014
[5-(5-Chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine hydrochloride salt Pigmented villonodular synovitis/giant cell tumor of the tendon sheath Daiichi Sankyo, Inc.;399 Thornall Street;Edison, New Jersey, 08837 February 14, 2014
Bruton’s tyrosine kinase inhibitor Chronic lymphocytic leukemia Celgene Corporation;400 Connell Drive, Suite 700;Berkeley Heights, New Jersey, 07922 February 10, 2014
DNA plasmid vector (pCK-HGFX7) expressing human hepatocyte growth factor Amyotrophic lateral sclerosis ViroMed Co., Ltd.;2221 Peachtree RD, NE, Suite D258;Atlanta, Georgia, 30309 February 6, 2014
Re188 P2045 somatostatin analog Small cell lung cancer Andarix Pharmaceuticals;141 Powderhouse Blvd, Suite 1;Somerville, Massachusetts, 02144 February 6, 2014
T cell receptor (TCR) peptide (BV5S2, BV6S5, BV13S1) vaccine Pediatric multiple sclerosis Immune Response BioPharma, Inc.;244 5th Avenue, Suite 1727;New York, 10001 February 6, 2014
Engineered variant of recombinant human fibroblast growth factor 19 Primary biliary cirrhosis NGM Biopharmaceuticals, Inc.;630 Gateway Blvd;South San Francisco, California, 94080 February 6, 2014
NorLeu3-Angiotensin(1-7) [NorLeu3-A(1-7)] Dermal injury due to nuclear/radiation incident US Biotest, Inc.;231 Bonetti Dr.,, Suite 240;San Luis Obispo, California, 93401 January 31, 2014
(tisagenlecleucel) Autologous T Cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 Acute lymphoblastic leukemia Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 January 31, 2014
Brentuximab vedotin Patients with diffuse large B-cell lymphoma Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 January 31, 2014
Autologous T Cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 Acute lymphoblastic leukemia Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 January 31, 2014
Cobimetinib Metastatic Melanoma Genentech, Inc.;1 DNA Way, MS 241A;South San Francisco, California, 94080 January 31, 2014
Flubendazole Lymphatic filariasis Janssen Research and Development, LLC;920 Route 202 South;Raritan, New Jersey, 08869 January 23, 2014
Ethanolamine Esophageal varices QOL Medical, LLC;4445 North Highway A1A, 241;Vero Beach, Florida, 32963 January 17, 2014
Allogeneic motor neuron progenitor cells derived from human embryonic stem cells Amyotrophic lateral sclerosis Caladrius Biosciences;110 Allen Road, 2nd Floor;Basking Ridge, New Jersey, 07920 January 17, 2014
Eculizumab Prevention of delayed graft function after renal transplantation Alexion Pharmaceuticals, Inc.;352 Knotter Drive;Cheshire, Connecticut, 06410 January 10, 2014
Enchochleate amphotericin B Leishmaniasis Aquarius Biotechnologies, Inc.;2037 West Carroll Avenue;Chicago, Illinois, 60612 January 10, 2014
Emicizumab-kxwh Hemophilia A Genentech;1 DNA Way;South San Francisco, California, 94080 January 10, 2014
Emicizumab Hemophilia A Genentech;1 DNA Way;South San Francisco, California, 94080 January 10, 2014
Factor VIII mimetic bispecific antibody Hemophilia A Genentech;1 DNA Way;South San Francisco, California, 94080 January 10, 2014