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
Autologous mononuclear cells derived from human cord blood Leukomalacia BrainRepair UG (haftungsbeschränkt) October 14, 2016
Venetoclax Diffuse Large B-cell Lymphoma AbbVie Ltd October 14, 2016
A non-covalent trimer of tumour necrosis factor fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragment format Soft tissue sarcoma Philogen S.p.A. October 14, 2016
Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 Diffuse Large B-cell Lymphoma Novartis Europharm Limited October 14, 2016
Xenon Ischaemia reperfusion injury associated with cardiac arrest Neuroprotexeon Ltd October 14, 2016
Crenolanib besylate Soft tissue sarcoma Arog Pharmaceuticals Europe Ltd October 14, 2016
Chemically modified human recombinant sulfamidase Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) Swedish Orphan Biovitrum AB (publ) October 14, 2016
Atezolizumab Small cell lung cancer (SCLC) Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 October 13, 2016
Vorinostat Metastatic Melanoma Qameleon Therapeutics;Moslaan 32, 1433 WJ;Kudelstaart October 13, 2016
Combination of an inactivated purified rabies virus (IPRV) and the PIKA adjuvant (Poly I:C, kanamycin and calcium) Hepatocellular Carcinoma Yisheng US Biopharma, Inc.;11 Firstfield Road, Suite A;Gaithersburg, Maryland, 20878 October 13, 2016
Ledipasvir/sofosbuvir Chronic hepatitis C virus (HCV) infection in pediatric patients Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 October 12, 2016
Liposomal vinorelbine Cutaneous T-cell lymphoma Taiwan Liposome Company, Ltd.;11F-1, No. 3, Yuanqu Street;Taipei City October 12, 2016
Vibrio alginolyticus collagenase Advanced Dupuytren's disease (Dupuytren's contracture) Fidia Farmaceutici S.p.A.;Via Ponte della Fabbrica, 3/A;Abano Terme PD October 12, 2016
Pembrolizumab Nasopharyngeal carcinoma Merck, Sharp & Dohme Corp., a subsidiary of Merck & Co.;1 Merck Drive;Whitehouse Station, New Jersey, 08889 October 11, 2016
Ibudilast Amyotrophic lateral sclerosis MediciNova, Inc.;4275 Executive Square, Suite 650;La Jolla, California, 92037 October 6, 2016
Recombinant human monoclonal antibody against human complement component C5a Graft Versus Host Disease Alexion Pharmaceuticals, Inc.;100 College Street;New Haven, Connecticut, 06510 October 6, 2016
Tacrolimus granules for oral suspension Prevention of rejection in kidney, liver or heart transplant in pediatric patients Astellas Pharma Global Development, Inc.;1 Astellas Way;Northbrook, Illinois, 60062 October 4, 2016
Autologous CD4+/CD8+ T cells expressing a CD19 chimeric antigen receptor (CAR) and a truncated epidermal growth factor receptor. Chronic lymphocytic leukemia Juno Therapeutics, Inc.;400 Dexter Avenue North, Suite 1200;Seattle, Washington, 98109 October 4, 2016
Posaconazole Invasive aspergillosis Merck, Sharp & Dohme Corp.;1 Merck Drive;Whitehouse Station, New Jersey, 08889 October 4, 2016
Atrasentan Focal segmental glomerulosclerosis AbbVie Inc.;Regulatory Affairs, 1 North Waukegan Road;North Chicago, Illinois, 60044 September 29, 2016
Recombinant adeno-associated virus serotype 8 vector encoding human glucose-6-phosphatase-alpha (G6Pase or G6PC) Von Gierke disease Dimension Therapeutics;840 Memorial Drive;Cambridge, Massachusetts, 02139 September 28, 2016
Wharton’s jelly mesenchymal stem cells Graft Versus Host Disease Midwest Stem Cell Therapy Center;University of Kansas Medical Center, 3901 Rainbow Boulevard;Kansas City, Kansas, 66160 September 27, 2016
Autologous dendritic cell/tumor antigens Glioblastoma China Medical University Hospital;No. 2, Yude Road, North District; September 27, 2016
Tenofovir disoproxil fumarate Prevention of mother to child transmission of hepatitis B virus Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 September 27, 2016
Methylprednisolone oral suspension Adrenal insufficiency in pediatric patients Generic Specialty Pharma;Unit 10, Block 3, City North Business Campus;Stamullin September 19, 2016