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
Bimagrumab Inclusion Body Myositis Novartis Pharmaceuticals Corp.;One Health Plaza;East Hanover, New Jersey, 07936 June 18, 2012
CNDO-109-activated allogeneic natural killer cells Acute myeloid leukemia Fortress Biotech, Inc.;2 Gansevoort Street, 9th Floor;New York, New York, 10014 June 18, 2012
Perhexiline maleate Moderate to severe symptomatic hypertrophic cardiomyopathy Heart Metabolics, Limited.;88 Harcourt Street, Dublin 2; June 14, 2012
Patisiran Amyloidosis Alnylam Pharmaceuticals, Inc.;300 Third Street;Cambridge, Maryland, 02142 June 7, 2012
Patisiran Transthyretin-mediated amyloidosis Alnylam Pharmaceuticals, Inc.;300 Third Street;Cambridge, Maryland, 02142 June 7, 2012
Recilisib sodium Acute Radiation Syndrome Acute radiation syndrome Onconova Therapeutics, Inc.;375 Pheasant Run;Newtown, Pennsylvania, 18940 June 1, 2012
(E)-4-carboxystyryl-4-chlorobenzyl-sulfone, sodium salt Prevention of acute radiation toxicity, also known as Acute Radiation Syndrome (ARS) Onconova Therapeutics, Inc.;375 Pheasant Run;Newtown, PA, 18940 June 1, 2012
Autologous Engineered Skin Substitute Hospitalized patients with deep partial and full thickness burns requiring grafting Amarantus BioScience Holdings, Inc.;655 Montgomery St., Suite 900;San Francisco, California, 94111 June 1, 2012
Bortezomib Mantle cell lymphoma Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 May 30, 2012
Heat Shock Protein (hsp60) antigen For use in type 1 diabetic mellitus patients with residual beta-cell function AndromedA Biotech, LTD;42 Hayarkon St.;Yavne 81227 May 21, 2012
Extract of sea cucumber, sea sponge, shark fin, sea urchin, and sargassum grass Multiple myeloma Unicorn Pacific Corporation;No. 1 Monmount Road, Bellevue Estate;Port Vila May 14, 2012
Eribulin mesylate Soft tissue sarcoma Eisai Inc.;155 Tice Blvd;Woodcliff Lake, New Jersey, 07677 May 14, 2012
Eribulin mesylate Advanced soft tissue sarcoma Eisai, Inc.;100 Tice Blvd;Woodcliff Lake, NJ, 07677 May 14, 2012
Alisertib T-cell lymphoma Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 May 14, 2012
Copper histidine Menkes syndrome Stephen G. Kaler, M.D.;Head, Unit on Human Copper Metabolism, 10 Center Drive-Rm 10N313, MSC 1853;Bethesda, Maryland, 20892 May 14, 2012
Eptifibatide and iloprost Purpura fulminans Thrombologic;COBIS/Ole Maaloesvej 3, 2200 Copenhagen; April 20, 2012
Autologous CD34+ hematopoietic stem cells tranduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA Andrenoleukodystrophy bluebird bio, Inc.;60 Binney Street;Cambridge, Massachusetts, 02142 April 19, 2012
N,N’-bis(2-mercaptoethyl)isophthalamide (NBMI) Mercury toxicity NBMI Science LLC;c/o Philip Lee Solicitors, 119 Burnside Drive;Nicholasville April 6, 2012
Ferumoxytol For use in magnetic resonance imaging to assess, and monitor treatment of solid tumor malignancies previously diagnosed in pediatric patients (age 16 years and younger) ArsNova Partners, LLC;38 Frederick Street;Newton, Massachusetts, 02460 April 6, 2012
Alpha1-proteinase inhibitor (human) Cystic fibrosis Grifols Therapeutics, Inc.;8368 US 70 BUS Hwy W.;Clayton, North Carolina, 27520 April 6, 2012
Adenovirus containing a human FAS-c gene Glioma Vascular Biogenics Ltd;6 Jonathan Netanyahu St;Or Yehuda April 2, 2012
Carisbamate West syndrome SK Life Science, Inc.;22-10 Route 208 South;Fair Lawn, New Jersey, 07410 March 16, 2012
O2-(2,4-dinitrophenyl) 1-[(4-ethoxycarbonyl)piperazin-1-yl]diazen-1-ium-1,2-diolate Acute myeloid leukemia JSK Therapeutics, Inc.;85 Fort Douglas Boulevard, P. O. Box 58986;Salt Lake City, Utah, 84158 March 16, 2012
Dehydrated alcohol Trigeminal neuralgia Luitpold Pharmaceuticals, Inc.;One Luitpold Drive, P. O. Box 9001;Shirley, New York, 11967 March 16, 2012
2,2′-{2-[1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid (ixazomib citrate) Systemic light chain (AL) amyloidosis Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 March 9, 2012