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
Human reovirus type 3 Dearing strain Ovarian cancer Oncolytics Biotech (UK) Limited March 19, 2015
5,10,15,20-tetrakis(2,6-difluoro-3-N-methylsulfamoylphenyl)bacteriochlorin Biliary tract cancer Luzitin S.A March 19, 2015
Trientine tetrahydrochloride Wilson disease gmp-orphan SA March 19, 2015
Recombinant human monoclonal antibody binding to vascular adhesion protein-1 Primary sclerosing cholangitis Biotie Therapies Corp March 19, 2015
Roneparstat Multiple myeloma Leadiant Biosciences, Inc.;Via Motta 2, CH-6850 Mendrisio; March 19, 2015
Melphalan flufenamide Multiple myeloma Oncopeptides AB March 19, 2015
Autologous adipose tissue-derived stromal vascular fraction cells Systemic scleroderma Assistance Publique Hôpitaux de Marseille March 19, 2015
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with aLAMB3-encoding retroviral vector Dystrophic epidermolysis bullosa Chiesi Farmaceutici S.p.A. March 19, 2015
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with aCOL7A1-encoding retroviral vector Dystrophic epidermolysis bullosa Chiesi Farmaceutici S.p.A. March 19, 2015
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with aCOL17A1-encoding retroviral vector Dystrophic epidermolysis bullosa Chiesi Farmaceutici S.p.A. March 19, 2015
Tremelimumab Malignant pleural mesothelioma MedImmune, LLC;One MedImmune Way;Gaithersburg, Maryland, 20878 March 18, 2015
Voretigene neparvovec-rzyl Inherited retinal dystrophy due to biallelic RPE65 gene mutations Spark Therapeutics, Inc.;3737 Market Street, Suite 1300;Philadelphia, Pennsylvania, 19104 March 18, 2015
Adeno-associated viral vector type 2 expressing human recombinant retinal pigment epithelial 65KDa protein gene Inherited retinal dystrophy due to biallelic RPE65 gene mutations Spark Therapeutics, Inc.;3737 Market Street, Suite 1300;Philadelphia, Pennsylvania, 19104 March 18, 2015
Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg](CH3COO) Amyotrophic lateral sclerosis Alsonex Pty Ltd;210 Alice Street, OLD, 4000;Brisbane March 9, 2015
Acetylcysteine effervescent tablets for oral solution Preventing hepatic injury from acetaminophin overdose Arbor Pharmaceuticals, Inc.;6 Concourse Parkway, Suite 1800;Atlanta, Georgia, 30328 February 24, 2015
Andexanet alfa For reversing the anticoagulant effect of direct or indirect factor Xa inhibitors in patients experiencing a serious uncontrolled bleeding event or who require urgent or emergent surgery Portola Pharmaceuticals;270 East Grand Avenue;South San Francisco, California, 94080 February 23, 2015
Bovine lactoferrin Necrotizing enterocolitis Metrodora Therapeutics, LLC;2502 Avenue I;Brooklyn, New York, 11210 February 23, 2015
Rituximab Pemphigus Genentech, Inc.;1 DNA Way;South San Francisco, California February 23, 2015
Naltrexone Postherpetic neuralgia Allodynic Therapeutics LLC;1785 NE 123rd Street;North Miami, Florida, 33181 February 23, 2015
Polidocanol Congenital venous malformations Provensis Ltd;5 Fleet Place, London EC4M 7RD; February 19, 2015
Bovine lactoferrin Prevention of late-onset sepsis in very low birth weight infants Metrodora Therapeutics, LLC;2502 Avenue I;Brooklyn, New York, 11210 February 19, 2015
Recombinant human glutamate oxaloacetate transaminase 1 Glioma Dr. Regenold GmbH February 12, 2015
Mazindol Narcolepsy HAC Pharma February 12, 2015
Sevuparin sodium Sickle cell anemia Modus Therapeutics AB February 12, 2015
N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4-methylpiperazin-1yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide TrkA-positive, TrkB-positive, TrkC-positive, ROS1-positive, or ALK-positive colorectal cancer Ignyta, Inc.;11095 Flintkote Avenue, Suite D;San Diego, CA, 92121 February 12, 2015