It’s only fair to share… Today, the U.S. Food and Drug Administration granted approval to Givlaari (givosiran) for the treatment of adult patients with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood). “This buildup …
Category «Breakthrough Therapy Designations»
FDA approves third oncology drug Rozlytrek (entrectinib) that targets a key genetic driver of cancer, rather than a specific type of tumor
It’s only fair to share… FDA approves third oncology drug Rozlytrek (entrectinib) that targets a key genetic driver of cancer, rather than a specific type of tumor FDA also approves drug for second indication in a type of lung cancer The U.S. Food and Drug Administration today granted accelerated approval to Rozlytrek (entrectinib), a treatment for …
Ritlectinib, PF 06651600
It’s only fair to share… Ritlectinib PF-06651600 CAS 1792180-81-4 C₁₅H₁₉N₅O, 285.34, UNII-2OYE00PC25 1-((2S,5R)-5-((7H-Pyrrolo[2,3-d]pyrimidin-4-yl)amino)-2-methylpiperidin-1-yl)prop-2-en-1-one 1-[(2S,5R)-2-Methyl-5-(7H-pyrrolo[2,3-d]pyrimidin-4-ylamino)-1-piperidinyl]-2-propen-1-one malonate PF-06651600 malonate CAS: 2140301-97-7 (malonate) Chemical Formula: C18H23N5O5 Molecular Weight: 389.412 PHASE 2 alopecia areata, rheumatoid arthritis, Crohn’s disease, and ulcerative colitis. PF-06651600 is a potent and selective JAK3 inhibitor. PF-06651600 is a potent and low clearance compound with demonstrated in vivo …
ブレキサノロン , Brexanolone, Allopregnanolone
It’s only fair to share… Brexanolone 318.501 g/mol, C21H34O2 CAS: 516-54-1 ブレキサノロン MFCD00003677 Pregnan-20-one, 3-hydroxy-, (3α,5α)- Pregnan-20-one, 3-hydroxy-, (3α,5α)- [ACD/Index Name] S39XZ5QV8Y TU4383000 UNII:S39XZ5QV8Y (1S,2S,7S,11S,14S,15S,5R,10R)-14-acetyl-5-hydroxy-2,15-dimethyltetracyclo[8.7.0.0<2,7>.0<11,15>]heptadecane (+)-3a-Hydroxy-5a-pregnan-20-one (+)-3α-Hydroxy-5α-pregnan-20-one (3α,5α)-3-Hydroxypregnan-20-one [ACD/IUPAC Name] 10446 3211363 [Beilstein] 3a-Hydroxy-5a-pregnan-20-one The U.S. Food and Drug Administration today approved Zulresso (brexanolone) injection for intravenous (IV) use for the treatment of postpartum depression (PPD) in adult women. …
Tagraxofusp タグラクソフスプ
It’s only fair to share… MGADDVVDSS KSFVMENFSS YHGTKPGYVD SIQKGIQKPK SGTQGNYDDD WKGFYSTDNK YDAAGYSVDN ENPLSGKAGG VVKVTYPGLT KVLALKVDNA ETIKKELGLS LTEPLMEQVG TEEFIKRFGD GASRVVLSLP FAEGSSSVEY INNWEQAKAL SVELEINFET RGKRGQDAMY EYMAQACAGN RVRRSVGSSL SCINLDWDVI RDKTKTKIES LKEHGPIKNK MSESPNKTVS EEKAKQYLEE FHQTALEHPE LSELKTVTGT NPVFAGANYA AWAVNVAQVI DSETADNLEK TTAALSILPG IGSVMGIADG AVHHNTEEIV AQSIALSSLM VAQAIPLVGE LVDIGFAAYN FVESIINLFQ VVHNSYNRPA YSPGHKTRPH MAPMTQTTSL KTSWVNCSNM IDEIITHLKQ PPLPLLDFNN LNGEDQDILM ENNLRRPNLE AFNRAVKSLQ NASAIESILK NLLPCLPLAT AAPTRHPIHI KDGDWNEFRR …
PF-06650833
It’s only fair to share… PF-06650833 1-{[(2S,3S,4S)-3-ethyl-4-fluoro-5-oxopyrrolidin-2-yl]methoxy}-7-methoxyisoquinoline-6-carboxamide CAS 1817626-54-2 Chemical Formula: C18H20FN3O4 Molecular Weight: 361.3734 Originator Pfizer Class Anti-inflammatories; Antirheumatics Mechanism of Action Interleukin-1 receptor-associated kinase inhibitors Phase II Rheumatoid arthritis Phase I Lupus vulgaris 01 Aug 2018 Pfizer completes a phase II trial in Rheumatoid arthritis (Treatment-experienced) in USA, Ukraine, Taiwan, Serbia, Russia, Romania, Poland, Mexico, South Korea, Georgia, Bosnia-Herzegovina, …
Beclabuvir
It’s only fair to share… BMS-791325, Beclabuvir IN PHASE 2 for Hepatitis C (HCV) An NS5B inhibitor. BMS-791325 preferably is CAS 958002-33-0 958002-36-3 (as hydrochloride) C36 H45 N5 O5 S, 659.838 Cycloprop(d)indolo(2,1-a)(2)benzazepine-9-carboxamide, 12-cyclohexyl-N-((dimethylamino)sulfonyl)-4b,5,5a,6-tetrahydro-3-methoxy-5a-((3-methyl-3,8-diazabicyclo(3.2.1)oct-8-yl)carbonyl)-, (4bS,5aR)- (4bS,5aR)-12-Cyclohexyl-N-(dimethylsulfamoyl)-3-methoxy-5a-((3-methyl-3,8-diazabicyclo(3.2.1)oct-8-yl)carbonyl)-4b,5,5a,6-tetrahydrocyclopropa(d)indolo(2,1-a)(2)benzazepine-9-carboxamide (4bS,5aR)-12-Cyclohexyl-N-(dimethylsulfamoyl)-3-methoxy-5a-((3-methyl-3,8-diazabicyclo(3.2.1)oct-8-yl)carbonyl)-4b,5,5a,6-tetrahydrocyclopropa(d)indolo(2,1-a)(2)benzazepine-9-carboxamide (1aR,12bS)-8-Cyclohexyl-N-(dimethylsulfamoyl)-11-methoxy-1a-[(3-methyl-3,8-diazabicyclo[3.2.1]oct-8-yl)carbonyl]-1,1a,2,12b-tetrahydrocyclopropa[d]indolo[2,1-a][2]benzazepine-5-carboxamide Cycloprop [d] indolo [2, 1 -a] [2]benzazepine-5-carboxamide, 8-cyclohexyl-N- [(dimethylamino)sulfonyl]-l,la,2,12b-tetrahydro-ll-methoxy-la-[(3-methyl-3,8- diazabicyclo[3.2.1]oct-8-yl)carbonyl]-, (laR,12bS)- Bristol-Myers Squibb (Originator) RNA-Directed RNA Polymerase (NS5B) …
Lanadelumab, ラナデルマブ
It’s only fair to share… (Heavy chain) EVQLLESGGG LVQPGGSLRL SCAASGFTFS HYIMMWVRQA PGKGLEWVSG IYSSGGITVY ADSVKGRFTI SRDNSKNTLY LQMNSLRAED TAVYYCAYRR IGVPRRDEFD IWGQGTMVTV SSASTKGPSV FPLAPSSKST SGGTAALGCL VKDYFPEPVT VSWNSGALTS GVHTFPAVLQ SSGLYSLSSV VTVPSSSLGT QTYICNVNHK PSNTKVDKRV EPKSCDKTHT CPPCPAPELL GGPSVFLFPP KPKDTLMISR TPEVTCVVVD VSHEDPEVKF NWYVDGVEVH NAKTKPREEQ YNSTYRVVSV LTVLHQDWLN GKEYKCKVSN KALPAPIEKT ISKAKGQPRE PQVYTLPPSR EEMTKNQVSL TCLVKGFYPS DIAVEWESNG QPENNYKTTP PVLDSDGSFF LYSKLTVDKS RWQQGNVFSC SVMHEALHNH YTQKSLSLSP G (Light …
Patisiran
It’s only fair to share… Patisiran Sense strand: GUAACCAAGAGUAUUCCAUdTdT Anti-sense strand: AUGGAAUACUCUUGGUUACdTdT RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA (G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1), ALN-18328, 6024128 , ALN-TTR02 , GENZ-438027 , SAR-438037 , 50FKX8CB2Y (UNII code) for RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA(G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1) Nucleic Acid Sequence Sequence Length: 42, 21, 2112 a 7 c 7 g 4 t 12 umultistranded (2); modified CAS 1420706-45-1 Treatment of Amyloidosis, …
FDA approves first treatment Azedra (iobenguane I 131) for rare adrenal tumors
It’s only fair to share… Iobenguane I 131 FDA approves first treatment for rare adrenal tumors The U.S. Food and Drug Administration today approved Azedra (iobenguane I 131) injection for intravenous use for the treatment of adults and adolescents age 12 and older with rare tumors of the adrenal gland (pheochromocytoma or paraganglioma) …