Tag «orphan drug»

Nanatinostat

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 Phase ISolid tumours No development …

Piflufolastat F 18 injection, Dcfpyl F-18

Piflufolastat F 18 injection Dcfpyl F-18 CAS 207181-29-0 PLAIN F 1423758-00-2 WITHOUT RADIO LABEL C18 H23 F N4 O8, 441.4 L-Glutamic acid, N-[[[(1S)-1-carboxy-5-[[[6-(fluoro-18F)-3-pyridinyl]carbonyl]amino]pentyl]amino]carbonyl]- 2-(3-{1-carboxy-5-[(6-[18F]fluoro-pyridine-3-carbonyl) amino]-pentyl}ureido)-pentanedioic acid Other Names N-[[[(1S)-1-Carboxy-5-[[[6-(fluoro-18F)-3-pyridinyl]carbonyl]amino]pentyl]amino]carbonyl]-L-glutamic acid [18F]DCFPyl Dcfpyl F-18 (18F)Dcfpyl UNII-3934EF02T7 18F-DCFPyL 3934EF02T7 Progenics Pharmaceuticals, Inc. APPROVED 5/26/2021 fda, Pylarify For positron emission tomography imaging of prostate-specific membrane antigen-positive lesions in men with prostate cancer For positron emission tomography …

Sotorasib

Sotorasib 6-fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-(4-methyl-2-propan-2-ylpyridin-3-yl)-4-[(2S)-2-methyl-4-prop-2-enoylpiperazin-1-yl]pyrido[2,3-d]pyrimidin-2-one AMG 510 AMG-510 AMG510 Formula C30H30F2N6O3 CAS 2296729-00-3 Mol weight 560.5944 FDA APPROVED, 2021/5/28 Lumakras Antineoplastic, Non-small cell lung cancer (KRAS G12C-mutated) ソトラシブ (JAN); 2296729-00-3 (racemate) 4-((S)-4-Acryloyl-2-methylpiperazin-1-yl)-6-fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-(2-isopropyl-4-methylpyridin-3-yl)pyrido[2,3-d]pyrimidin-2(1H)-one 6-fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-(4-methyl-2-propan-2-ylpyridin-3-yl)-4-[(2S)-2-methyl-4-prop-2-enoylpiperazin-1-yl]pyrido[2,3-d]pyrimidin-2-one Sotorasib [INN] 6-Fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-(4-methyl-2-propan-2-ylpyridin-3-yl)-4-((2S)-2-methyl-4-prop-2-enoylpiperazin-1-yl)pyrido(2,3-d)pyrimidin-2-one Sotorasib (1M)-6-Fluoro-7-(2-fluoro-6-hydroxyphenyl)-1-[4-methyl-2-(propan-2-yl)pyridin-3-yl]-4-[(2S)-2-methyl-4-(prop-2-enoyl)piperazin-1-yl]pyrido[2,3-d]pyrimidin-2(1H)-one C30H30F2N6O3 : 560.59 [2296729-00-3] Sotorasib is an inhibitor of the RAS GTPase family. The molecular formula is C30H30F2N6O3, and the molecular weight is 560.6 g/mol. …

Infigratinib phosphate

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 CAS 1310746-10-1 FREE form 872511-34-7 Mol …

Ibrexafungerp citrate

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; Triterpenes Mechanism of ActionBeta-1,3-D glucan …

Loncastuximab tesirine

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 musculus monoclonal RB4v1.2 κ-chain, dimer, …

Devimistat

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 as pyruvate dehydrogenase and alpha-ketoglutarate …

Naxitamab

(Heavy chain) QVQLVESGPG VVQPGRSLRI SCAVSGFSVT NYGVHWVRQP PGKGLEWLGV IWAGGITNYN SAFMSRLTIS KDNSKNTVYL QMNSLRAEDT AMYYCASRGG HYGYALDYWG QGTLVTVSSA STKGPSVFPL APSSKSTSGG TAALGCLVKD YFPEPVTVSW NSGALTSGVH TFPAVLQSSG LYSLSSVVTV PSSSLGTQTY ICNVNHKPSN TKVDKRVEPK SCDKTHTCPP CPAPELLGGP SVFLFPPKPK DTLMISRTPE VTCVVVDVSH EDPEVKFNWY VDGVEVHNAK TKPREEQYNS TYRVVSVLTV LHQDWLNGKE YKCKVSNKAL PAPIEKTISK AKGQPREPQV YTLPPSRDEL TKNQVSLTCL VKGFYPSDIA VEWESNGQPE NNYKTTPPVL DSDGSFFLYS KLTVDKSRWQ QGNVFSCSVM HEALHNHYTQ KSLSLSPGK (Light chain) EIVMTQTPAT LSVSAGERVT ITCKASQSVS NDVTWYQQKP GQAPRLLIYS ASNRYSGVPA …

Lumasiran

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 g 14 umultistranded (2); modified …

Berotralstat

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 is a synthetic, once-daily, small …