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 adipose tissue derived mesenchymal stem cells Buerger’s disease Biostar Stem Cell Research Institute;305 Lotte IT Castle 2,, 550-1 Gasan-dong;Seoul May 4, 2016
Acyclovir Herpetic keratitis Cumulus Pharmaceuticals LLC;1712 Pioneer Avenue, Suite 1377;Cheyenne, Wyoming, 82001 May 3, 2016
Fancalen (lentiviral vector containing the Fanconi anemia-A (FANCA) gene Fanconi Anemia Consorcio Centro de Investigaci�n Biom�dica en Red, M.P.;Montforte de Lemos, 3-5 Pabellon 11;Madrid May 2, 2016
Combinatorial regimen of LV305 (lentiviral vector encoding NY-ESO-1 gene) and G305 (NY-ESO-1 recombinant protein plus GLA-SE) Soft tissue sarcoma Immune Design Corp.;1616 Eastlake Ave. E, Suite 310;Seattle, Washington, 98102 May 2, 2016
Autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor Mantle cell lymphoma Kite Pharma, Inc.;2225 Colorado Avenue;Santa Monica, California, 90404 April 28, 2016
N-carboxymethyl-glycyl-L-threonyl-L-histidyl-L-3,3-diphenylalanyl-L-piperidincarboxy-3-yl-L-arginyl-L-S-methylthio-cystyl-L-arginyl-L-tryptophyl-aminohexanyl-N-carboxamidomethyl-glycine N-hexadecylamide Ataxia-Telangiectasia QRC Consultants Ltd April 28, 2016
Resiquimod Cutaneous T-cell lymphoma Galderma R&D April 28, 2016
S-acetyl-(S)-4′-phosphopantetheine, calcium salt Pantothenate-kinase-associated neurodegeneration Acies Bio d.o.o. April 28, 2016
(1E,6E)-1,7-bis(3,4-dimethoxyphenyl)-4-cyclobutylmethyl-1,6-heptadiene-3,5-dione Spinal muscular atrophy Coté Orphan Consulting UK Limited April 28, 2016
Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, Ser-Gly-Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-Asp-Glu-Leu-Val-Arg-His-His-Asn-Met-His-Gln-Arg-Asn-Met-Thr-Lys-Leu and Pro-Gly-Cys-Asn-Lys-Arg-Tyr-Phe-Lys-Leu-Ser-His-Leu-Gln-Met-His-Ser-Arg-Lys-His-Thr-Gly Acute myeloid leukaemia SELLAS Life Sciences Group UK, Limited April 28, 2016
Autologous dermal fibroblasts genetically modified ex vivo with a lentiviral vector containing the humanCOL7A1gene Dystrophic epidermolysis bullosa Intrexon Actobiotics N.V. April 28, 2016
Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, Ser-Gly-Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-Asp-Glu-Leu-Val-Arg-His-His-Asn-Met-His-Gln-Arg-Asn-Met-Thr-Lys-Leu and Pro-Gly-Cys-Asn-Lys-Arg-Tyr-Phe-Lys-Leu-Ser-His-Leu-Gln-Met-His-Ser-Arg-Lys-His-Thr-Gly Malignant pleural mesothelioma SELLAS Life Sciences Group UK, Limited April 28, 2016
Autologous stromal vascular cell fraction from adipose tissue Systemic scleroderma Cytori Ltd April 28, 2016
Fluocinolone acetonide Non-infectious uveitis Campharm Ltd April 28, 2016
Humanised recombinant IgG4 anti-human tau antibody Progressive supranuclear palsy AbbVie Ltd April 28, 2016
Human/murine chimeric monoclonal antibody against endoglin Soft tissue sarcoma Tracon Pharma Limited April 28, 2016
Recombinant adeno-associated viral vector serotype 9 carrying the gene for the human E6-AP ubiquitin protein ligase Angelman syndrome Voisin Consulting S.A.R.L. April 28, 2016
Recombinant human cerebral dopamine neurotrophic factor Amyotrophic lateral sclerosis Herantis Pharma Plc April 28, 2016
2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol methanesulfonate Acute myeloid leukaemia Celgene Europe Limited April 28, 2016
Brigatinib Non-small cell lung cancer ARIAD Pharmaceuticals, Inc.;26 Landsdowne Street;Cambridge, Massachusetts, 02139 April 28, 2016
Antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA Leber congenital amaurosis ProQR Therapeutics IV BV April 28, 2016
Brincidofovir Prevention of cytomegalovirus disease Chimerix UK Ltd April 28, 2016
Cannabidiol Graft Versus Host Disease Richardson Associates Regulatory Affairs Ltd April 28, 2016
Combination of 4-hydroxyandrostenedione, Serenoa serrulata fruit extract and alpha lipoic acid Multiple symmetric lipomatosis Dr. Regenold GmbH April 28, 2016
6-{[(1S)-1-phenylethyl]amino}-3-(propan-2-yl)-1,2,3,4 tetrahydropyrimidine-2,4-dione Symptomatic obstructive hypertrophic cardiomyopathy MyoKardia, Inc.;333 Allerton Avenue;South San Francisco, California, 94080 April 27, 2016