Tag «Orphan drug designation»

IMIPRIDONE

It’s only fair to share… IMIPRIDONE CAS No. : 1616632-77-9 Molecular Weight, 386.4964 Related CAS #: 41276-02-2 (TIC10 isomer)   1616632-77-9 (free base)   1638178-82-1 (HCl)   1777785-71-3 (HBr)   2007141-57-1 (2HBr) TIC 10, 0NC 201, OP 10 Synonym: ONC201; ONC 201; ONC-201; NSC350625; NSC-350625; NSC 350625; TIC10; TIC 10; TIC-10; TRAIL inducing compound 10; imipridone 7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one 2,4,6,7,8,9-Hexahydro-4-((2-methylphenyl)methyl)-7-phenylmethyl)imidazo)(1,2-a)pyrido(3,4-e)pyrimidin-5(1H)-one ONC-201 Dihydrochloride C24H28Cl2N4O 459.4 UNII-53VG71J90J …

Tebentafusp-tebn

It’s only fair to share… Tebentafusp-tebn IMCGP100 UNIIN658GY6L3E CAS number1874157-95-5 FDA APPROVED 1/25/2022, Kimmtrak, To treat unresectable or metastatic uveal melanoma Immunocore Limited T cell receptor α chain (synthetic human) fusion protein with T cell receptor β chain (synthetic human) fusion protein with immunoglobulin, anti-(human CD3 antigen) (synthetic scFv fragment) Protein Sequence Sequence Length: 695, 500, 195 Sequence: …

ZY 19489, MMV 253

It’s only fair to share… ZY 19489, MMV 253 C24 H32 FN9, 465.5 CAS 1821293-40-6 MMV253, GTPL10024, MMV674253 N-(4-cyclopropyl-5-fluoro-6-methylpyridin-2-yl)-5-((3R)-2-((1,5-dimethyl-1H-pyrazol-3-yl)amino)-3,4-dimethylpiperazin-1-yl)pyrimidin-2-amine 2-N-(4-cyclopropyl-5-fluoro-6-methylpyridin-2-yl)-5-[(3R)-3,4-dimethylpiperazin-1-yl]-4-N-(1,5-dimethylpyrazol-3-yl)pyrimidine-2,4-diamine N2-(4-Cyclopropyl-5-fluoro-6-methyl-2-pyridinyl)-5-[(3R)-3,4-dimethyl-1-piperazinyl]-N4-(1,5-dimethyl-1H-pyrazol-3-yl)-2,4-pyrimidinediamine (R)-N2-(4-Cyclopropyl-5-fluoro-6-methylpyridin-2-yl)-N4-(1,5-dimethyl-1H-pyrazol-3-yl)-5-(3,4-dimethylpiperazin-1-yl)pyrimidine-2,4-diamine SYN IN 201721031453 The invention relates to triaminopyrimidine compd. of formula I, pharmaceutically acceptable salts thereof, hydrates, solvates, polymorphs, optically active forms thereof, in solid state forms useful for preventing or treating malaria.  The invention also relates …

Avalglucosidase alfa

It’s only fair to share… QQGASRPGPR DAQAHPGRPR AVPTQCDVPP NSRFDCAPDK AITQEQCEAR GCCYIPAKQG LQGAQMGQPW CFFPPSYPSY KLENLSSSEM GYTATLTRTT PTFFPKDILT LRLDVMMETE NRLHFTIKDP ANRRYEVPLE TPRVHSRAPS PLYSVEFSEE PFGVIVHRQL DGRVLLNTTV APLFFADQFL QLSTSLPSQY ITGLAEHLSP LMLSTSWTRI TLWNRDLAPT PGANLYGSHP FYLALEDGGS AHGVFLLNSN AMDVVLQPSP ALSWRSTGGI LDVYIFLGPE PKSVVQQYLD VVGYPFMPPY WGLGFHLCRW GYSSTAITRQ VVENMTRAHF PLDVQWNDLD YMDSRRDFTF NKDGFRDFPA MVQELHQGGR RYMMIVDPAI SSSGPAGSYR PYDEGLRRGV FITNETGQPL IGKVWPGSTA FPDFTNPTAL AWWEDMVAEF HDQVPFDGMW IDMNEPSNFI RGSEDGCPNN ELENPPYVPG VVGGTLQAAT …

BELUMOSUDIL

It’s only fair to share… BELUMOSUDIL C26H24N6O2 MW 452.5 911417-87-3, SLx-2119, KD-025, KD 025, WHO 11343 2-[3-[4-(1H-indazol-5-ylamino)quinazolin-2-yl]phenoxy]-N-propan-2-ylacetamide 2-(3-(4-(lH-indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide Belumosudil mesylate KD025 mesylate 2109704-99-4 Belumosudil mesylate is an orally available rho kinase 2 (ROCK 2) inhibitor being developed at Kadmon. In 2020, the drug candidate was submitted for a new drug application (NDA) in the U.S., under a …

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 …

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 …

NIROGACESTAT

It’s only fair to share… NIROGACESTAT (2S)-2-[[(2S)-6,8-difluoro-1,2,3,4-tetrahydronaphthalen-2-yl]amino]-N-[1-[1-(2,2-dimethylpropylamino)-2-methylpropan-2-yl]imidazol-4-yl]pentanamide 489.6 g/mol, C27H41F2N5O CAS 1290543-63-3 PF-03084014, 1290543-63-3, PF-3084014, 865773-15-5 QZ62892OFJ UNII:QZ62892OFJ UNII-QZ62892OFJ нирогацестат [Russian] [INN] نيروغاسيستات [Arabic] [INN] 尼罗司他 [Chinese] [INN] ニロガセスタット; orphan drug designation in June 2018 for the treatment of desmoid tumors, and with a fast track designation  Nirogacestat, also known as PF-03084014, is a potent and selective gamma secretase (GS) inhibitor with potential antitumor activity. PF-03084014 …

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 …

FDA approves novel treatment Oxbryta (voxelotor) to target abnormality in sickle cell disease

It’s only fair to share… Today, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older. “Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million …