Category «Orphan Drug Status»

Lurbinectedin

It’s only fair to share… Lurbinectedin (1’R,6R,6aR,7R,13S,14S,16R)-5-(Acetyloxy)-2′,3′,4′,6,6a,7,9′-decahydro-8,14-dihydroxy-6′,9-dimethoxy-4,10,23-trimethyl-spiro(6,16-(epithiopropaneoxymethano)-7.13-imino-12H-1,3-dioxolo[7,8]soquino[3,2-b][3]benzazocine-20,1′-[1H]pyrido[3,4-b]indol]-19-one Molecular Weight 784.87 Formula C41H44N4O10S CAS No. 497871-47-3 (Lurbinectedin); Chemical Name Spiro[6,16-(epithiopropanoxymethano)-7,13-imino-12H-1,3-dioxolo[7,8]isoquino[3,2-b][3]benzazocine-20,1′-[1H]pyrido[3,4-b]indol]-19-one, 5-(acetyloxy)-2′,3′,4′,6,6a,7,9′,13,14,16-decahydro-8,14-dihydroxy-6′,9-dimethoxy-4,10,23-trimethyl-, (1’R,6R,6aR,7R,13S,14S,16R)- (9CI) fda approved , 6/15/2020 , ZEPZELCA, Pharma Mar S.A. To treat metastatic small cell lung cancer Drug Trials Snapshot Research Code:PM-01183; PM-1183 MOA:RNA polymerase inhibitor Indication:Ovarian cancer; Breast cancer; Non small cell lung cancer (NSCLC) лурбинектедин لوربينيكتيدين 芦比替定 …

Ansuvimab-zykl

It’s only fair to share… Ansuvimab-zykl FDA APPROVED, 12/21/2020, EBANGA To treat ebola https://www.fda.gov/drugs/drug-safety-and-availability/fda-approves-treatment-ebola-virus The U.S. Food and Drug Administration approved Ebanga (Ansuvimab-zykl), a human monoclonal antibody, for the treatment for Zaire ebolavirus (Ebolavirus) infection in adults and children. Ebanga blocks binding of the virus to the cell receptor, preventing its entry into the cell. Zaire ebolavirus is one …

Lumasiran

It’s only fair to share… The molecular formula of lumasiran sodium is C530H669F10N173O320P43S6Na43 and the molecular weight is 17,286 Da. lumasiran CAS 1834610-13-7 FDA APPROVED, 11/23/2020, Oxlumo To treat hyperoxaluria type 1 Press Release Drug Trials Snapshot RNA, (Gm-sp-Am-sp-Cm-Um-Um-Um-(2′-deoxy-2′-fluoro)C-Am-(2′-deoxy-2′-fluoro)U-(2′-deoxy-2′-fluoro)C-(2′-deoxy-2′-fluoro)C-Um-Gm-Gm-Am-Am-Am-Um-Am-Um-Am), 3′-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]-1,12,19,25-tetraoxo-16-oxa-13,20,24-triazanonacos-1-yl]-4-hydroxy-2-pyrrolidinyl]methyl hydrogen phosphate], complex with RNA (Um-sp-(2′-deoxy-2′-fluoro)A-sp-Um-Am-Um-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)C-(2′-deoxy-2′-fluoro)C-Am-Gm-Gm-Am-(2′-deoxy-2′-fluoro)U-Gm-(2′-deoxy-2′-fluoro)A-Am-Am-Gm-Um-Cm-sp-Cm-sp-Am) (1:1) Nucleic Acid Sequence Sequence Length: 44, 23, 2115 a 8 c 7 …

Setmelanotide

It’s only fair to share… Setmelanotide Ac-Arg-Cys(1)-D-Ala-His-D-Phe-Arg-Trp-Cys(1)-NH2   Molecular FormulaC49H68N18O9S2 Average mass1117.309 Da N-acetyl-L-arginyl-L-cysteinyl-D-alanyl-L-histidyl-D-phenylalanyl-L-arginyl-L-tryptophyl-L-cysteinamide (2->8)-disulfide 1,2-Dithia-5,8,11,14,17,20-hexaazacyclotricosane-4-carboxamide, 22-[[(2S)-2-(acetylamino)-5-[(diaminomethylene)amino]-1-oxopentyl]amino]-10-[3-[(diaminomethylene)amino]propyl]-16-(1H-imidazol-5-ylmeth yl)-7-(1H-indol-3-ylmethyl)-19-methyl-6,9,12,15,18,21-hexaoxo-13-(phenylmethyl)-, (4R,7S,10S,13R,16S,19R,22R)- [ACD/Index Name] 10011 920014-72-8 [RN] Imcivree [Trade name] N2-acetyl-L-arginyl-L-cysteinyl-D-alanyl-L-histidyl-D-phenylalanyl-L-arginyl-Ltryptophyl- L-cysteinamide, cyclic (2-8)-disulfide N7T15V1FUY RM-493, BIM-22493 UNII-N7T15V1FUY сетмеланотид [Russian] [INN] سيتميلانوتيد [Arabic] [INN] 司美诺肽 [Chinese] [INN] (4R,7S,10S,13R,16S,19R,22R)-22-[[(2S)-2-acetamido-5-(diaminomethylideneamino)pentanoyl]amino]-13-benzyl-10-[3-(diaminomethylideneamino)propyl]-16-(1H-imidazol-5-ylmethyl)-7-(1H-indol-3-ylmethyl)-19-methyl-6,9,12,15,18,21-hexaoxo-1,2-dithia-5,8,11,14,17,20-hexazacyclotricosane-4-carboxamide FDA 11/25/2020, Imcivree, To treat obesity and the control of hunger associated with pro-opiomelanocortin deficiency, a rare disorder that causes severe obesity …

Berotralstat

It’s only fair to share… Berotralstat CAS  1809010-50-1 DIHCl 1809010-52-3 Molecular Formula, C30-H26-F4-N6-O, Molecular Weight, 562.5684 1-(3-(Aminomethyl)phenyl)-N-(5-((R)-(3-cyanophenyl)((cyclopropylmethyl)amino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide 1H-Pyrazole-5-carboxamide, 1-(3-(aminomethyl)phenyl)-N-(5-((R)-(3-cyanophenyl)((cyclopropylmethyl)amino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)- To treat patients with hereditary angioedema FDA APPROVED 12/4/2020, Orladeyo, 110MG CAPSULE 0RAL New Drug Application (NDA): 214094 Company: BIOCRYST PHARMACEUTICALS INC New Drug Application (NDA): 214094 Company: BIOCRYST PHARMACEUTICALS INC Berotralstat Hydrochloride 1-[3-(Aminomethyl)phenyl]-N-(5-{(1R)-(3-cyanophenyl)[(cyclopropylmethyl)amino]methyl}-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide dihydrochloride C30H26F4N6O▪2HCl : 635.48 [1809010-52-3] Berotralstat, also known as BCX-7353, is a kallikrein inhibitor. BCX7353 …

Pemigatinib

It’s only fair to share…   Pemigatinib INCB054828 Formula C24H27F2N5O4 CAS 1513857-77-6 2379919-96-5  HCL Mol weight 487.4991 2020/4/17FDA APPROVED, PEMAZYRE 佩米替尼 [Chinese] [INN] 3-(2,6-Difluoro-3,5-dimethoxyphenyl)-1-ethyl-8-(morpholinomethyl)-1,3,4,6-tetrahydro-2H-pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one 2H-Pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one, 3-(2,6-difluoro-3,5-dimethoxyphenyl)-1-ethyl-1,3,4,7-tetrahydro-8-(4-morpholinylmethyl)- 3-(2,6-difluoro-3,5-dimethoxyphenyl)-1-ethyl-8-(morpholin-4-ylmethyl)-1,3,4,7-tetrahydro-2H-pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one OriginatorIncyte Corporation DeveloperIncyte Corporation; Innovent Biologics ClassAntineoplastics; Ethers; Fluorobenzenes; Morpholines; Pyridines; Pyrimidinones; Pyrroles; Small molecules Mechanism of ActionType 1 fibroblast growth factor receptor antagonists; Type 3 fibroblast growth factor receptor …

Bulevirtide acetate

It’s only fair to share…   Bulevirtide acetate (N-Myristoyl-glycyl-L-threonyl-L-asparaginyl-L-leucyl-L-seryl-L-valyl-Lprolyl-L-asparaginyl-L-prolyl-L-leucyl-glycyl-L-phenylalanyl-L-phenylalanyl-L-prolyl-L-aspartyl-L-histidyl-Lglutaminyl-L-leucyl-L-aspartyl-L-prolyl-L-alanyl-L-phenylalanyl-glycyl-L-alanyl-L-asparaginyl-L-seryl-Lasparaginyl-L-asparaginyl-Lprolyl-L-aspartyl-L-tryptophanyl-L-aspartyl-L-phenylalanyl-L-asparaginyl-L-prolylL-asparaginyl-L-lysyl-L-aspartyl-L-histidyl-L-tryptophanyl-L-prolyl-L-glutamyl-L-alanyl-L-asparaginyl-L-lysylL-valylglycinamide, acetate salt. molecular formula C248H355N65O72, molecular mass is 5398.9 g/mol ブレビルチド酢酸塩; APROVED 2020/7/31, EU, Hepcludex MYR GmbH Antiviral, Entry inhibitor   Disease Hepatitis delta virus infection Bulevirtide is a 47-amino acid peptide with a fatty acid, a myristoyl residue, at the N-terminus and an amidated C-terminus. The active substance …

Teprotumumab-trbw

It’s only fair to share… Tepezza (teprotumumab-trbw) Company: Horizon Therapeutics plc Date of Approval: January 21, 2020 Treatment for: Thyroid Eye Disease Labels for BLA 761143 UNIIY64GQ0KC0A CAS number1036734-93-6 R-1507 / R1507 / RG-1507 / RG1507 / RO-4858696 / RO-4858696-000 / RO-4858696000 / RO4858696 / RO4858696-000 / RV-001 / RV001 Tepezza (teprotumumab-trbw) is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor 1 receptor (IGF-1R) for the treatment of active thyroid eye …

Brilliant blue G , ブリリアントブルーG ,

It’s only fair to share…   Brilliant blue G FDA 2019, 12/20/2019, TISSUEBLUE, New Drug Application (NDA): 209569 Company: DUTCH OPHTHALMIC, PRIORITY; Orphan OPQ recommends APPROVAL of NDA 209569 for commercialization of TissueBlue (Brilliant Blue G Ophthalmic Solution), 0.025% Neuroprotectant sodium;3-[[4-[[4-(4-ethoxyanilino)phenyl]-[4-[ethyl-[(3-sulfonatophenyl)methyl]azaniumylidene]-2-methylcyclohexa-2,5-dien-1-ylidene]methyl]-N-ethyl-3-methylanilino]methyl]benzenesulfonate Formula C47H48N3O7S2. Na CAS 6104-58-1 Mol weight 854.0197 ブリリアントブルーG, C.I. Acid Blue 90 UNII-M1ZRX790SI M1ZRX790SI 6104-58-1 Brilliant Blue G Derma …

Avapritinib, アバプリチニブ , авапритиниб , أفابريتينيب ,

It’s only fair to share… Avapritinib BLU-285, BLU285 Antineoplastic, Tyrosine kinase inhibitor アバプリチニブ авапритиниб [Russian] [INN] أفابريتينيب [Arabic] [INN] (1S)-1-(4-fluorophenyl)-1-[2-[4-[6-(1-methylpyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]piperazin-1-yl]pyrimidin-5-yl]ethanamine (1S)-1-(4-Fluorophenyl)-1-(2-{4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl}-5-pyrimidinyl)ethanamine 10613 1703793-34-3 [RN] 513P80B4YJ 5-Pyrimidinemethanamine, α-(4-fluorophenyl)-α-methyl-2-[4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl]-, (αS)- (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-1-yl)pyrimidin-5-yl)ethan-1-amine (αS)-(4-fluorophenyl)-α-methyl-2-[4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl]-5-pyrimidinemethanamine Formula C26H27FN10 CAS 1703793-34-3 Mol weight 498.558 No. Drug Name Active Ingredient Approval Date FDA-approved use on approval date* 1. Ayvakit avapritinib 1/9/2020 To treat adults with unresectable or metastatic …