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Decitabine (i.e., 5-aza-2′-deoxycytidine), sold under the brand name Dacogen among others, acts as a nucleic acid synthesis inhibitor. [4] It is a medication for the treatment of myelodysplastic syndromes, a class of conditions where certain blood cells are dysfunctional, and for acute myeloid leukemia (AML). [5] Chemically, it is a cytidine ...
Azacitidine and decitabine are marketed as Vidaza and Dacogen respectively. Azacitidine is the first drug to be approved by FDA for treating MDS and has been given orphan drug status. [ 2 ] [ 3 ] Procaine is a DNA-demethylating agent with growth-inhibitory effects in human cancer cells. [ 4 ]
A hypomethylating agent (or demethylating agent [1]) is a drug that inhibits DNA methylation: the modification of DNA nucleotides by addition of a methyl group.Because DNA methylation affects cellular function through successive generations of cells without changing the underlying DNA sequence, treatment with a hypomethylating agent is considered a type of epigenetic therapy.
Decitabine/cedazuridine was approved for medical use in the United States and Canada in July 2020, [7] [8] [9] [12] and in the European Union in September 2023. [6]The U.S. Food and Drug Administration (FDA) granted the application of decitabine combined with cedazuridine priority review and orphan drug designation.
Azacitidine is a chemical analogue of the nucleoside cytidine, which is present in DNA and RNA.It is thought to have antineoplastic activity via two mechanisms – at low doses, by inhibiting of DNA methyltransferase, causing hypomethylation of DNA, [16] and at high doses, by its direct cytotoxicity to abnormal hematopoietic cells in the bone marrow through its incorporation into DNA and RNA ...
Icatibant received orphan drug status in Australia, the EU, Switzerland, and the US for the treatment of hereditary angioedema (HAE). [7]In the EU, the approval by the European Commission (July 2008) allows Jerini to market Firazyr in the European Union's 27 member states, as well as Switzerland, Liechtenstein and Iceland, making it the first product to be approved in all EU countries for the ...
Tibolone, sold under the brand name Livial among others, is a medication which is used in menopausal hormone therapy and in the treatment of postmenopausal osteoporosis and endometriosis. [ 1 ] [ 9 ] [ 10 ] [ 11 ] The medication is available alone and is not formulated or used in combination with other medications. [ 12 ]
Hydroxycarbamide, also known as hydroxyurea, is an antimetabolite medication used in sickle-cell disease, essential thrombocythemia, chronic myelogenous leukemia, polycythemia vera, and cervical cancer. [4] [5] In sickle-cell disease it increases fetal hemoglobin and decreases the number of attacks. [4] It is taken by mouth. [4]