Category «Orphan Drug Status»

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 …

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 …