Category «orphan drug»

Nanatinostat

It’s only fair to share… Nanatinostat Tractinostat CHR-3996, CHR 3996, VRx 3996, C20H19FN6O2, 394.41 CAS 1256448-47-1 2-[(1α,5α,6α)-6-[[(6-Fluoro-2-q 2-[(1R,5S,6R)-6-{[(6-fluoroquinolin-2-yl)methyl]amino}-3-azabicyclo[3.1.0]hexan-3-yl]-N-hydroxypyrimidine-5-carboxamide 2-[(1R,5S,6s)-6-{[(6-Fluoro-2-quinolinyl)methyl]amino}-3-azabicyclo[3.1.0]hex-3-yl]-N-hydroxy-5-pyrimidinecarboxamide 5-Pyrimidinecarboxamide, 2-[(1R,5S)-6-[[(6-fluoro-2-quinolinyl)methyl]amino]-3-azabicyclo[3.1.0]hex-3-yl]-N-hydroxy- Chroma Therapeutics Ltd. (Originator) OriginatorChroma Therapeutics DeveloperChroma Therapeutics; Viracta Therapeutics ClassAmides; Antineoplastics; Pyrimidines; Quinolines; Small molecules Mechanism of ActionHistone deacetylase inhibitors Orphan Drug StatusYes – Post-transplant lymphoproliferative disorder; Plasmablastic lymphoma; T-cell lymphoma Phase IILymphoma Phase I/IIMultiple myeloma …

Pegcetacoplan

It’s only fair to share… Sequence: 1ICVWQDWGAH RCTXK Sequence: 1ICVWQDWGAH RCTXK Sequence Modifications Type Location Description terminal mod. Lys-15 C-terminal amide terminal mod. Lys-15′ C-terminal amide bridge Cys-2 – Cys-12 disulfide bridge, dimer bridge Lys-15 – Lys-15′ covalent bridge, dimer bridge Cys-2′ – Cys-12′ disulfide bridge, dimer uncommon Oaa-14 – uncommon Oaa-14′ – Pegcetacoplan ペグセタコプラン; …

Infigratinib phosphate

It’s only fair to share… Infigratinib phosphate FDA APPR Truseltiq 2021/5/28 インフィグラチニブリン酸塩; 3-(2,6-dichloro-3,5-dimethoxyphenyl)-1-[6-[4-(4-ethylpiperazin-1-yl)anilino]pyrimidin-4-yl]-1-methylurea;phosphoric acid BGJ 398 BGJ-398 BGJ398 NVP-BGJ398 WHO 10032 Product Ingredients INGREDIENT UNII CAS INCHI KEY Infigratinib acetate 03D0789NYP 1310746-17-8 XHCQHOGMMJKLRU-UHFFFAOYSA-N Infigratinib hydrochloride WY8VD4RV77 1310746-15-6 VBAIJSJSFCXDJB-UHFFFAOYSA-N Infigratinib mesylate E223Z0KWCC 1310746-12-3 BXJJFNXYWJLBOS-UHFFFAOYSA-N Infigratinib phosphate 58BH47BV6S 1310746-10-1 GUQNHCGYHLSITB-UHFFFAOYSA-N International/Other BrandsTruseltiq (BridgeBio Pharma, Inc.)   Formula C26H31Cl2N7O3. H3PO4 …

Ibrexafungerp citrate

It’s only fair to share… Ibrexafungerp citrate アイブレキサフンジェルプクエン酸塩; Formula C44H67N5O4. C6H8O7 cas 1965291-08-0 free 1207753-03-4 Mol weight 922.1574 Brexafemme, fda approved 2021, 2021/6/1 Antifungal, Cell wall biosynthesis inhibitor, Treatment of invasive fungal infections due to Candida spp. or Aspergillus spp., vulvovaginal candidiasis SCY-078 citrate, MK-3118; SCY-078, WHO 10597 UNII-M4NU2SDX3E M4NU2SDX3E (1R,5S,6R,7R,10R,11R,14R,15S,20R,21R)-21-[(2R)-2-amino-2,3,3-trimethylbutoxy]-5,7,10,15-tetramethyl-7-[(2R)-3-methylbutan-2-yl]-20-(5-pyridin-4-yl-1,2,4-triazol-1-yl)-17-oxapentacyclo[13.3.3.01,14.02,11.05,10]henicos-2-ene-6-carboxylic acid;2-hydroxypropane-1,2,3-tricarboxylic acid Originator Merck & Co; SCYNEXIS Class Antifungals; Glycosides; …

Idecabtagene vicleucel

It’s only fair to share…   Idecabtagene vicleucel CAS 2306267-75-2 STN: BLA 125736 An autologous T lymphocyte-enriched cell transduced ex vivo with an anti-BCMA CAR lentiviral vector encoding a chimeric antigen receptor CAR, comprising a CD8 hinge and TM domain, 4-1BB costimulatory domain and CD3ζ signaling domain, targeting human B cell maturation antigen for cancer immunotherapy (Celgene …

Loncastuximab tesirine

It’s only fair to share…   Loncastuximab tesirine ZYNLONTA FDA APPROVED 2021/4/23 https://www.adcreview.com/wp-content/uploads/2019/06/15_ICML_Chiara-Tarantelli-ADCT_402_preclinical.pdf Formula C6544H10048N1718O2064S52 Exact mass 147387.9585 CAS 1879918-31-6 Efficacy Antineoplasitc, Anti-CD19 antibody   Disease Diffuse large B-cell lymphoma not otherwise specified [DS:H02434] Comment Antibody-drug conjugate Treatment of hematological cancers ロンカスツキシマブテシリン; ADCT-402, ADCX 19 Immunoglobulin G1, anti-(human CD19 antigen) (human-Mus musculus monoclonal RB4v1.2 γ1-chain), disulfide with human-Mus …

Fosdenopterin hydrobromide

It’s only fair to share… Fosdenopterin hydrobromide FDA APPR 2021/2/26, NULIBRY BBP-870/ORGN001 a cyclic pyranopterin monophosphate (cPMP) substrate replacement therapy, for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A. ホスデノプテリン臭化水素酸塩水和物; Formula C10H14N5O8P. 2H2O. HBr CAS 2301083-34-9DIHYDRATE Mol weight 480.1631 2301083-34-9 (1R,10R,12S,17R)-5-amino-11,11,14-trihydroxy-14-oxo-13,15,18-trioxa-2,4,6,9-tetraza-14λ5-phosphatetracyclo[8.8.0.03,8.012,17]octadeca-3(8),4-dien-7-one;dihydrate;hydrobromide 1,3,2-DIOXAPHOSPHORINO(4′,5′:5,6)PYRANO(3,2-G)PTERIDIN-10(4H)-ONE, 8-AMINO-4A,5A,6,9,11,11A,12,12A-OCTAHYDRO-2,12,12-TRIHYDROXY-, 2-OXIDE, HYDROBROMIDE, HYDRATE (1:1:2), (4AR,5AR,11AR,12AS)- CYCLIC PYRANOPTERIN MONOPHOSPHATE MONOHYDROBROMIDE DIHYDRATE …

TRIFAROTENE

It’s only fair to share… Trifarotene CAS 895542-09-3 3”-Tert-butyl-4′-(2-hydroxyethoxy)-4”-(pyrrolidin-1-yl)(1,1′:3′,1”)terphenyl-4-carboxylic acid 3′-[3-tert-butyl-4-(pyrrolidin-1-yl)phenyl]-4′-(2-hydroxyethoxy)-[1,1′-biphenyl]-4-carboxylic acid [1,1′:3′,1”-Terphenyl]-4-carboxylic acid, 3”-(1,1-dimethylethyl)-4′-(2-hydroxyethoxy)-4”-(1-pyrrolidinyl)- 0J8RN2W0HK 4′-(2-Hydroxyethoxy)-3”-(2-methyl-2-propanyl)-4”-(1-pyrrolidinyl)-1,1′:3′,1”-terphenyl-4-carboxylic acid UNII-0J8RN2W0HK, Galderma Research & Development 459.5766 C29 H33 N O4 CD-5789 CD5789 трифаротен [Russian] [INN] تريفاروتين [Arabic] [INN] 曲法罗汀 [Chinese] [INN] Trifarotene, sold under the brand name Aklief, is a medication for the topical treatment of acne vulgaris in those nine years of age and older.[1] It …

Devimistat

It’s only fair to share…   Devimistat CPI-613 Molecular Weight 388.59 Formula C₂₂H₂₈O₂S₂ CAS No. 95809-78-2 SMILES O=C(O)CCCCC(SCC1=CC=CC=C1)CCSCC2=CC=CC=C2 phase III, hematological cancer 6,8-Bis(benzylsulfanyl)octanoic acid Octanoic acid, 6,8-bis[(phenylMethyl)thio]- Octanoic acid, 6,8-bis((phenylmethyl)thio)- Rafael Pharmaceuticals (formerly Cornerstone Pharmaceuticals), a subsidiary of Rafael Holdings, is developing devimistat, the lead candidate from a program of thioctans and their derivatives that act …

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 …