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
Inactivated purified rabies virus (IPRV) and the PIKA adjuvant (Poly I:C, kanamycin and calcium) Rabies Yisheng US Biopharma, Inc.;11 Firstfield Road, Suite A;Gaithersburg, Maryland, 20878 December 29, 2016
Ganaxolone Fragile X syndrome Marinus Pharmaceuticals;170 North Radnor Chester Road, Suite 250;Radnor, Pennsylvania, 19087 December 28, 2016
Pembrolizumab Merkel cell cancer Merck, Sharp, and Dohme Corp.;1 Merck Access Road;New Jersey, 08889 December 28, 2016
Silmitasertib Bile duct cancer Senhwa Biosciences, Inc.;No. 205-1, Peihsin Road, Section 3;New Taipei City December 22, 2016
CD123 x CD3 dual affinity re-targeting (DART®) protein Acute myeloid leukemia MacroGenics, Inc.;9704 Medical Center Drive;Rockville, Maryland, 20850 December 22, 2016
CD123 x CD3 dual affinity re-targeting (DART�) protein Acute myeloid leukemia MacroGenics, Inc.;9704 Medical Center Drive;Rockville, Maryland, 20850 December 22, 2016
Nivolumab Stomach Cancer Bristol-Myers Squibb Co.;5 Research Parkway;Wallingford, Connecticut, 06492 December 20, 2016
Nivolumab and Ipilimumab Stomach Cancer Bristol-Myers Squibb Co.;5 Research Parkway;Wallingford, Connecticut, 06492 December 20, 2016
Eflornithine Pancreatic cancer Cancer Prevention Pharmaceutical, Inc.;1760 East River Road, Suite 250;Tucson, Arizona, 85718 December 14, 2016
3-aminopropyl-butyl phosphinic acid Succinic semialdehyde dehydrogenase deficiency Speragen, Inc.;13492 Research Boulevard, Suite 120-475;Austin, Texas, 78750 December 13, 2016
Tetrandrine Acute myeloid leukemia Escend Pharmaceuticals, Inc.;3475 Edison Way, Suite R;Menlo Park, California, 94025 December 13, 2016
68Ga-DOTA-pABzA-DIG-dPhe-Gln-Trp-Ala-Val-Gly-His-NHCH[(CH2-CH(CH3)2]2 Diagnosis of gastrointestinal stromal tumours Advanced Accelerator Applications December 12, 2016
Polatuzumab vedotin Diffuse Large B-cell Lymphoma Genentech, Inc.;1 DNA Way, MS# 355E;South San Francisco, California, 94080 December 12, 2016
Adeno-associated viral vector serotype 8 containing the humanCNGA3gene under the control of a cone arrestin promoter Achromatopsia Universitätsklinikum Tübingen (UKT) December 12, 2016
Adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes Retinitis pigmentosa Fondazione Telethon December 12, 2016
Propranolol Soft tissue sarcoma The Anticancer Fund December 12, 2016
Arsenic trioxide Graft Versus Host Disease Medsenic December 12, 2016
Cabiralizumab Tenosynovial giant cell tumour, localised and diffuse type TMC Pharma Services Ltd December 12, 2016
Ivosidenib Acute myeloid leukaemia QRC Consultants Ltd December 12, 2016
Pegylated recombinant human interleukin-10 Pancreatic Cancer Larode Ltd December 12, 2016
Avelumab Stomach Cancer Merck Serono Europe Limited December 12, 2016
Udenafil Functional single ventricle congenital heart disease Mapi Ireland Limited December 12, 2016
Dantrolene sodium Wolfram syndrome Alan Boyd Consultants Ltd December 12, 2016
Immune globulin (human) Myasthenia gravis Myasthenia gravis Grifols Therapeutics, Inc.;79 TW Alexander Drive;Research Triangle Park, North Carolina, 27709 December 12, 2016
Ibudilast Amyotrophic lateral sclerosis MediciNova (Europe) Limited December 12, 2016