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
40K PEGylated recombinant factor IX Hemophilia B Novo Nordisk, Inc.;100 College Road West;Princeton, 08540 March 18, 2013
Cell based therapeutic composed of allogeneic donor apoptotic cells Graft Versus Host Disease Enlivex Therapeutics Ltd.;POB 12167, Hadassah Ein Kerem;Jerusalem March 18, 2013
40K PEGylated recombinant factor IX Routine prophylactic administration for prevention of bleeding in patients with hemophilia B (Christmas disease) Novo Nordisk, Inc.;100 College Road West;Princeton, 08540 March 18, 2013
Chimeric monoclonal antibody against Claudin 6 Ovarian cancer Astellas Pharma Global Development, Inc.;1 Astellas Way;Northbrook, Illinois, 60062 March 18, 2013
Recombinant adeno-associated viral vector containing the humanretinoschisingene X-linked Juvenile Retinoschisis TMC Pharma Services Ltd March 12, 2013
His-His-Ile-Tyr-Leu-Gly-Ala-Val-Asn-Tyr-Ile-Tyr Retinal detachment ONL Therapeutics, Inc;1600 Huron Parkway, Second Floor;Ann Arbor, Michigan, 48109 March 11, 2013
Aerosolized beractant Respiratory distress syndrome Beena G. Sood, MD, MS;Wayne State University School of Medicine, Children's Hospital fo MI;Detroit, Michigan, 48201 March 11, 2013
C66H100N6O27 Hepatocellular Carcinoma GenSpera, Inc.;2511 North Loop 1604 W, Suite 204;San Antonio, Texas, 78258 March 11, 2013
AAV-G6Pase vector Glycogen storage disease type Ia GlyGenix Therapeutics, Inc.;15 Research Drive;Woodbridge, Connecticut, 06443 March 11, 2013
3-bromopyruvate Bile duct cancer Primocure Pharma, Inc.;1732 1st Avenue, #23222;New York, New York, 10128 March 5, 2013
Allogeneic ex-vivo expanded placental adherent stromal cells Aplastic anemia aplastic anemia Novella Clinical, Inc.;MATAM Advanced Technology Park, Haifa 31905; February 18, 2013
Minnelide Pancreatic Cancer Minneamrita Therapeutics, LLC;6 Hickory Court;Rock Island, Illinois, 61201 February 18, 2013
2-hydroxypropyl-B-cyclodextrin Niemann Pick disease, type C Vtesse, Inc.;700 Technology Square, Third Floor;Cambridge, Massachusetts, 02139 February 18, 2013
Enalapril maleate (powder for oral solution) Hypertension in pediatric patients Silvergate Pharmaceuticals, Inc.;5371 Gordon Way;Dublin, Ohio, 43017 January 30, 2013
[met5]-enkephalin Pancreatic Cancer TNI BioTech, Inc.;6701 Democracy Blvd, Suite 300;Bethesda, Maryland, 20817 January 24, 2013
10 synthetic peptides targeting 5 tumor asociated antigens Non-small cell lung cancer Orphan Synergy Europe Pharma (OSE Pharma);OSE Pharma Pepiniere Paris Sante Cochin, 29 bis rue du faubourg Saint Jacques;Paris January 23, 2013
Exon 45 specific phosphorothioate oligonucleotide Duchenne muscular dystrophy BioMarin Pharmaceutical, Inc.;105 Digital Drive;Novato, California, 94949 January 23, 2013
Exon 52 specific phosphorothiate oligonucleotide Duchenne muscular dystrophy BioMarin Pharmaceutical, Inc.;105 Digital Drive;Novato, California, 94949 January 23, 2013
Exon 55 specific phosphorothioate oligonucleotide Duchenne muscular dystrophy BioMarin Pharmaceutical, Inc.;105 Digital Drive;Novato, California, 94949 January 23, 2013
Exon 53 specific phosphorothioate oligonucleotide Duchenne muscular dystrophy BioMarin Pharmaceutical, Inc.;105 Digital Drive;Novato, California, 94949 January 23, 2013
Eflornithine plus sulindac Familial adenomatous polyposis Cancer Prevention Pharmaceuticals, Inc.;1760 East River Road, Suite 250;Tucson, Arizona, 85718 January 22, 2013
Apremilast Behçet’s Disease Celgene Corporation;86 Morris Avenue;Summit, New Jersey, 07901 January 17, 2013
Beloranib Prader-Willi syndrome Zafgen, Inc.;175 Portland Street, 4th Floor;Boston, Massachusetts, 02114 January 15, 2013
5-aminolevulinic acid Acute respiratory distress syndrome NX Development Corporation;1827 South Bayshore Lane;Miami, Florida, 33133 January 15, 2013
Efdispo ™ Ewings Sarcoma TDP Biotherapeutics, Inc.;113 Summer Walk Drive;Gaithersburg, Maryland, 20878 January 15, 2013