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
Adeno-associated virus delivered transgene of follistatin Inclusion Body Myositis Milo Biotechnology;2322 Delaware Drive;Cleveland Heights, Ohio, 44106 September 19, 2016
Antisense oligonucleotide inhibitor of the expression of superoxide dismutase 1 gene Amyotrophic lateral sclerosis Biogen;225 Binney Street;Cambridge, Massachusetts, 02142 September 19, 2016
Decitabine and tetrahydrouridine Sickle cell anemia EpiDestiny Inc.;7536 Royal Portrush Drive;Solon, Ohio, 44139 September 19, 2016
3-(5-amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride Diffuse Large B-cell Lymphoma Celgene Corporation;400 Connell Drive, Suite 7000;Berkeley Heights, New Jersey, 07922 September 19, 2016
Methylprednisolone oral suspension Adrenal insufficiency in pediatric patients Generic Specialty Pharma;Unit 10, Block 3, City North Business Campus;Stamullin September 19, 2016
ProTmune (ex vivo Programmed Mobilized Peripheral Blood) Graft Versus Host Disease Fate Therapeutics;3535 General Atomics Court, Suite 200;San Diego, California, 92121 September 19, 2016
Fixed dose combination of modafinil and flecainide Narcolepsy Theranexus S.A;86 Rue de Paris;Orsay September 19, 2016
Disulfide-stabilized single-chain variable region fragment of the anti-EGFR wild type/EGFRvIII dual specificity monoclonal antibody with variable fragments fused to a truncated version of the Pseudomonas exotoxin A, PE38, and KDEL Glioma Darell D. Bigner, MD, PhD;Duke University Medical Center, Oreuss Laboratory -Brain Tumor Research;Durham, North Carolina, 27710 September 19, 2016
Riluzole oral suspension Amyotrophic lateral sclerosis Italfarmaco SpA;54 Via dei Lavoratori;Cinisello Balsamo September 15, 2016
Emodepside Onchocerhiasis ("river blindness") caused by the filarial nematode onchocerca volvulus Bayer HealthCare Pharmaceuticals, Inc.;100 Bayer Boulevard, P. O. Box 915;Whippany, New Jersey, 07981 September 13, 2016
Poly(lactide-co-glycolide) carboxylated microparticle Malaria Cour Pharmaceutical Development Company, Inc.;428 S. Hillside Avenue;Elmhurst, Illinois, 60126 September 13, 2016
Terlipressin Ascites due to all etiologies except cancer Bio Vie Inc.;100 Cummings Center, Suite 247-C;Beverly, Massachusetts, 01915 September 8, 2016
Coversin; rEV675 Hemoglobinuria Akari Therapeutics Plc;24 West 40th Street, 8th Floor;New York, New York, 10018 September 8, 2016
Autologous CD4+/CD8+ T cells expressing a CD19 chimeric antigen receptor and a truncated epidermal growth factor receptor Acute lymphoblastic leukemia Juno Therapeutics, Inc.;400 Dexter Avenue North, Suite 1200;Seattle, Washington, 98109 September 7, 2016
6-fluoro-(18F)-L-3,4-dihydroxyphenylalanine A diagnostic for management of patients with Lesch-Nyhan syndrome Advanced Imaging Projects, LLC;7947 Brookside Ct.;Lake Worth, Florida, 33467 September 7, 2016
Brentuximab vedotin Primary cutaneous CD30-positive T-cell lymphoproliferative disorders Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 September 7, 2016
Rituximab and recombinant human hyaluronidase Diffuse Large B-cell Lymphoma Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 September 7, 2016
Cinacalcet Focal segmental glomerulosclerosis Amgen, Inc.;One Amgen Center Drive, Bldg 17, 1A-4-4, Mail Stop 17-1-B;Thousand Oaks, California, 91320 September 7, 2016
Mesylate synthetic small molecule inhibitor of HDAC and PI3K Nuclear protein in testis (NUT) midline carcinoma Curis, Inc.;4 Maguire Road;Lexington, Massachusetts, 02421 September 6, 2016
Cisplatin Anal cancer Privo Technologies;13 Bristol Street, Unit #1;Cambridge, Massachusetts, 02141 September 6, 2016
Bilayer, engineered collagen hydrogel-based skin graft composed of autologous keratinocytes and fibroblasts Partial deep dermal and full thickness burn wounds University Children's Hospital Zurich;Tissue Biology Research Unit, 7 August-Forel-Strasse;Z�rich September 6, 2016
4,5,6,7-tetrahydroisoxazolo(5,4-c)pyridin-3-ol Angelman syndrome Ovid Therapeutics;1460 Broadway;New York, New York, 10036 September 6, 2016
Dabrafenib and trametinib Papillary thyroid cancer Novartis Pharmaceuticals Corporation;1 Health Plaza, Building 337;East Hanover, New Jersey, 07936 September 1, 2016
Angiotensin (1-7) Recessive dystrophic epidermolysis bullosa (RDEB) Tarix Orphan, LLC;12 Bow Street;Cambridge, Massachusetts, 02138 September 1, 2016
Recombinant adeno-associated virus vector serotype 9 expressing human iduronate-2-sulfatase Hunter Syndrome Research Institute at Nationwide Children's Hospital;Center for Gene Therapy, 700 Childrens Drive;Columbus, Ohio, 43205 September 1, 2016