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
3-bromopyruvate Pancreatic Cancer PreScience Labs, LLC;9500 Reach Road;Potomac, Maryland, 20854 April 29, 2014
A live attenuated bioengineered Listeria monocytogenes cancer immunotherapy Stage II to IV invasive cervical carcinoma Advaxis, Inc.;305 College Road East;Princeton, New Jersey, 08540 April 29, 2014
(R)-1-(2,2-difluorobenzo [d][1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide Cystic fibrosis Vertex Pharmaceuticals Inc.;50 Northern Avenue;Boston, Massachusetts, 02210 April 24, 2014
Alvocidib Acute myeloid leukemia Tolero Pharmaceuticals, Inc.;2975 West Executive Parkway, Suite 320;Lehi, Utah, 84043 April 21, 2014
Allopregnanolone Status epilepticus Sage Therapeutics;215 First Street, Suite 220;Cambridge, Massachusetts, 02142 April 20, 2014
Ac-Ile-Cys-Val-Trp(1-Me)-Gln-Asp-Trp-Gly-Ala-His-Arg-Cys-Thr-AEEA-Lys-PEG 40KDa-Lys-AEEA-Thr-Cys-Arg-His-ALa-Gly-Trp-Asp-Gln-(1-Me)Trp-Val-Cys-Ile-Ac Hemoglobinuria Apellis Pharmaceuticals, Inc.;6400 Westwind Way, Suite A;Crestwood, Kentucky, 40014 April 20, 2014
Ciprofloxacin dry powder inhaler Bronchiectasis Bayer HealthCare Pharmaceuticals, Inc.;P. O. Box 1000;Montville, New Jersey, 07045 April 17, 2014
Benznidazole Chagas disease Chemo Research, S.L.;Edificio Nectar, c/Quintanapalla, 2, 3 planta;Madrid April 14, 2014
6-(1H-Indazol-6-yl)-N-(4-morpholinophenyl)imidazo-[1,2-alpha]pyrazin-8-amine bis-methanesulfonate Chronic lymphocytic leukemia Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 April 14, 2014
Anthrax Vaccine Adsorbed Anthrax Emergent BioDefense Operations Lansing LLC;300 Proessional Drive;Gaithersburg, Maryland, 20879 April 11, 2014
H-Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Arg-Lys-Leu-Ser-Ser-Ile-Glu- Ser-Asp-Val-OH Acute ischemic stroke patients presenting within 3 hours of symptom onset NoNO, Inc.;399 Bathurst Street, Suite 4W-435;Toronto April 11, 2014
Carbetocin Prader-Willi syndrome Ferring Pharmaceuticals, Inc.;4 Gatehall Drive, 3rd Floor;Parsippany, NJ, 07054 April 11, 2014
Carbetocin (nasal spray) Prader-Willi syndrome Ferring Pharmaceuticals, Inc.;4 Gatehall Drive, 3rd Floor;Parsippany, New Jersey, 07054 April 11, 2014
Laromustine Acute myeloid leukemia Nanotherapeutics, Inc.;13859 Progress Blvd, Suite 300;Alachua, Florida, 32615 March 27, 2014
L-asparaginase Acute myeloid leukemia ERYTECH Pharma S.A.;Batiment Adenine, 60, avenue Rockefeller;Lyon March 27, 2014
Autologous peripheral blood T lymphocytes transduced with retroviral vector containing anti CD19 CD28/CD3 zeta chimeric antigen receptor Diffuse large B-cell lymphoma Kite Pharma, Inc.;2225 Colorado Avenue;Santa Monica, California, 90404 March 27, 2014
4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide Diffuse large B-cell lymphoma AbbVie;Research and Development, 1 North Waukegan Road;North Chicago, Illinois, 60064 March 27, 2014
Adeno-associated viral vector serotype 8 containing the humanGUCY2Dgene Leber congenital amaurosis Fondazione Telethon March 26, 2014
(RS)-baclofen, naltrexone and D-sorbitol Charcot-Marie-Tooth disease Pharnext SA;11, rue des Peupliers, 92130 Issey les Moulineaux; March 17, 2014
Ad5/3-D24-granulocyte-macrophage colony stimulating factor (GMCSF)-encoding oncolytic adenovirus Ovarian cancer Targovax YO;Saukonpaadenranta 2;Helsinki March 17, 2014
Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 Chronic lymphocytic leukemia Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 March 14, 2014
Adeno associated virus with modified transthyretin and sequence encoding factor IX variant gene Hemophilia B Baxalta US, Inc.;One Baxter Way;Westlake Village, California, 91362 March 14, 2014
Fecal microbiota Recurrent Clostridium difficile infection (Clostridium difficile gastrointestinal disease) Rebiotx, Inc.;2660 Patton Road;Roseville, Minnesota, 55113 March 10, 2014
Cannabidiol; Lennox-Gastaut syndrome GW Pharma Ltd.;Porton Down Science Park, Salisbury, Wiltshire SP4 OJQ; February 27, 2014
2-[(1R,6R)-3-Methyl-6-(1-methylethenyl)-2-cyclohexen-1-yl]-5-pentyl-1,3-benzenediol Lennox-Gastaut syndrome GW Pharma Ltd.;Porton Down Science Park, Salisbury, Wiltshire SP4 OJQ; February 27, 2014