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
Synthetic human hepcidin Sickle cell anemia Cogas Pharma Limited November 18, 2016
Ascorbic acid Facioscapulohumeral muscular dystrophy Université de Montpellier November 18, 2016
Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Gly-Gly-Asp-Leu-Leu-Pro-Arg-Gly-Ser Huntington disease Dr Ulrich Granzer November 18, 2016
Budesonide IgA Nephropathy Pharmalink AB November 18, 2016
Vaccine consisting of 5 survivin peptides with different human leukocyte antigen restrictions Ovarian cancer Dr Ulrich Granzer November 18, 2016
Ibrutinib Graft Versus Host Disease Janssen-Cilag International NV November 18, 2016
L-selenomethionine Facioscapulohumeral muscular dystrophy Université de Montpellier November 18, 2016
Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Arg-Gly-Gly-Asp-Leu-Leu-Pro-Arg-Gly-Ser Huntington disease Dr Ulrich Granzer November 18, 2016
N-(5-(6-chloro-2,2-difluorobenzo[d][1,3]dioxol-5-yl)pyrazin-2-yl)-2-fluoro-6-methylbenzamide Acute pancreatitis Cogas Pharma Limited November 18, 2016
Particles comprised of methacrylic acid based co-polymer, cross-linked with a bi-functional cross-linker, purified to bind L-phenylalanine and L-phenylalanine containing peptides Hyperphenylalaninaemia MipSalus ApS November 18, 2016
R-azasetron besylate Sensorineural hearing loss Sensorion November 18, 2016
Recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein Aromatic amino acid decarboxylase deficiency Diamond BioPharm Limited November 18, 2016
Sodium benzoate N-acetylglutamate synthetase deficiency Lucane Pharma SA November 18, 2016
Valproic acid Diffuse Large B-cell Lymphoma Valcuria AB November 18, 2016
Zinc gluconate Facioscapulohumeral muscular dystrophy Université de Montpellier November 18, 2016
2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde Sickle cell anemia SynteractHCR Deutschland GmbH November 18, 2016
5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride X-linked adrenoleukodystrophy Minoryx Therapeutics S.L. November 18, 2016
Brincidofovir Smallpox Chimerix UK Ltd November 18, 2016
Allogeneic cytomegalovirus-specific cytotoxic T lymphocytes Cytomegalovirus infection in patients with impaired cell-mediated immunity Atara Biotherapeutics Ireland Limited November 18, 2016
Adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene Glycogen storage disease type Ia Pharma Gateway AB November 18, 2016
Human monoclonal antibody against activin A Fibrodysplasia ossificans progressiva Regeneron Ireland November 18, 2016
Allogeneic peripheral blood mononuclear cells incubated ex vivo with 16, 16-dimethyl prostaglandin E2 and dexamethasone Treatment in haematopoietic stem cell transplantation Fate Therapeutics Ltd November 18, 2016
Live-attenuated non-replicativePseudomonas aeruginosastrain expressing large T antigen of Merkel cell polyomavirus Merkel cell cancer APCure SAS November 18, 2016
Diazepam buccal soluble film Selected, refractory patients with epilepsy who are on stable regimens of antiepileptic drugs (AED) and who require intermittent use of diazepam to control bouts of increased seizure activity (acute repetitive seizures) Aquestive Therapeutics;30 Technology Drive South;Warren, New Jersey, 07059 November 10, 2016
Sargramostim Acute Radiation Syndrome Acute radiation syndrome Sanofi Genzyme;500 Kendall Street;Cambridge, Massachusetts, 02142 November 9, 2016