Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

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Country Product Indication Sponsor Date
3-pentylbenzeneacetic acid sodium salt Alström syndrome ProMetic Pharma SMT Limited January 12, 2017
Antroquinonol Pancreatic Cancer Biological Consulting Europe Ltd January 12, 2017
Monovalent replication-incompetent adenovirus serotype 26 (Ad26) vaccine, AD26.ZEBOV in combination with multivalent replication-defective Modified Vaccinia Ankara (MVA)-Bavarian Nordic (BN) vaccine, MVA-mBN226B Ebola Crucell Holland B.V.;Archimedesweg 4-6;Leiden January 11, 2017
Non-replicating recombinant adeno-associated viral vector expressing the human acid alpha-glucosidase gene Pompe disease Duke University Medical Center;Box 103856;Durham, North Carolina, 27710 January 11, 2017
Human single domain antibody-like protein inhibitor of the chemokine receptor type 4 Idiopathic pulmonary fibrosis AdAlta Limited;15/2 Park Drive;Bundoora January 11, 2017
4-methylpiperazine-1-carboxylic acid [1-(3-benzenesufonyl-1-phenethylallyl-carbamoyl)-2-phenylethyl] amide Chagas disease Center for Discovery & Innovation in Parasitic Diseases;University of CA, San Francisco, 9500 Gilman Drive;LaJolla, California, 92093 January 11, 2017
Ritonavir powder for oral suspension Use with other antiretroviral agents for the treatment of pediatric HIV-1 infection AbbVie Inc.;Regulatory Affairs, 1 Waukegan Road;North Chicago, Illinois, 60044 January 11, 2017
Protoplasmic protein matrix, omega-3 fatty oils, vitamins, minerals and trace elements, cell membrane matrix phospholipid, entracellular matrix, lipids, medium chain triglycerides, longer chain triglycerides, carbohydrates, probiotic, and phytonutrients Juvenile rheumatoid arthritis Leonard S. Girsh, MD;Immunopath Profile, Ind., P. O. Box 770805;Naples, Florida, 34107 January 11, 2017
Glecaprevir and pibrentasvir Pediatric patients with chronic hepatitis C virus infection AbbVie, Inc.;1 Waukegan Road;North Chicago, Illinois, 60044 January 9, 2017
Adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human alpha-L-iduronidase (hIDUA) gene Hurler syndrome Sangamo Therapeutics, Inc.;Point Richmond Tech Center, 501 Canal Boulevard;Richmond, California, 94804 January 9, 2017
Clenbuterol Pompe disease Dwight D. Koeberl, MD, PhD;Duke University Medical Center, Pediatrics Dept., Div. of Genetics;Durham, North Carolina, 27710 January 9, 2017
L-citrulline Acute respiratory distress syndrome Asklepion Pharmaceuticals, LLC;729 East Pratt Street, Suite 360;Baltimore, Maryland, 21202 January 9, 2017
Vancomycin HCL and Tobramycin sulfate multi-pack component of the Joint Purification Systems X7 Spacer Periprosthetic joint infection Joint Purification Systems LLC;c/o Cor Medical Ventures LLC, 215 S. Highway 101;Solana Beach, California, 92075 January 9, 2017
Echinomycin Graft Versus Host Disease OncoImmune, Inc.;502 E. Street, NE;Washington, District of Columbia, 20002 January 5, 2017
Celecoxib oral liquid suspension Pediatric juvenile idiopathic arthritis NuBioPharma, LLC;111 Dennis Drive, Suite 121;Sanford, North Carolina, 27330 January 5, 2017
Cytomegalovirus specific cytotoxic T lymphocytes Cytomegalovirus infection in severely immunocompromised patients ViraCyte, LLC;33 - 5th Avenue, NW, Suite 700;New Brighton, Minnesota, 55112 January 5, 2017
Ublituximab and phosphoinositide-3-kinase delta inhibitor Chronic lymphocytic leukemia TG Therapeutics, Inc.;2 Gansevoort Street, 9th Floor;New York, New York, 10014 January 5, 2017
(S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile Dystrophic epidermolysis bullosa Almirall, S.A.;Ctra. Laurea Miro, 408-410, Sant Feliu de Llobregat;Barcelona January 5, 2017
Emixustat Stargardt Disease Acucela Inc.;1301 2nd Avenue, Suite 4200;Seattle, Washington, 98101 January 4, 2017
Pyridoxal-5’ phosphate monohydrate (P5P) Seizures associated with pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency Medicure International Inc.;2-1250 Waverley Street;Winnipeg, Manitoba January 4, 2017
Pyridopyrimidinone SMN2 splicing modifier Spinal muscular atrophy Hoffmann-La Roche, Inc.;c/o Genentech, Inc., 1 DNA Way;South San Francisco, California, 94080 January 4, 2017
Humanized monoclonal antibody that blocks activity of complement component C5 Hemoglobinuria Alexion Pharmaceuticals, Inc.;55 Cambridge Parkway, Suite 800;Cambridge, Massachusetts, 02142 January 4, 2017
Lenalidomide Splenic marginal zone lymphoma Celgene Corporation;86 Morris Avenue;Summit, New Jersey, 07901 January 4, 2017
Lenalidomide Nodal marginal zone lymphoma Celgene Corporation;86 Morris Avenue;Summit, New Jersey, 07901 January 4, 2017
Pyridoxal-5� phosphate monohydrate (P5P) Seizures associated with pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency Medicure International Inc.;2-1250 Waverley Street;Winnipeg, Manitoba January 4, 2017