Tag «Orphan drug designation»

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 …

CK-101

It’s only fair to share…   CK-101, RX-518 CAS 1660963-42-7 MF C29 H28 F2 N6 O2 MW 530.57 2-Propenamide, N-[3-[2-[[2,3-difluoro-4-[4-(2-hydroxyethyl)-1-piperazinyl]phenyl]amino]-8-quinazolinyl]phenyl]- N-[3-[2-[[2,3-Difluoro-4-[4-(2-hydroxyethyl)piperazin-1-yl]phenyl]amino]quinazolin-8-yl]phenyl]acrylamide N-(3-(2-((2,3-Difluoro-4-(4-(2-hydroxyethyl)piperazin-1-yl)phenyl)amino)quinazolin-8-yl)phenyl)acrylamide EGFR-IN-3 UNII-708TLB8J3Y 708TLB8J3Y AK543910 Suzhou NeuPharma (Originator) Checkpoint Therapeutics Non-Small Cell Lung Cancer Therapy Solid Tumors Therapy PHASE 2 Checkpoint Therapeutics, Cancer, lung (non-small cell) (NSCLC), solid tumour RX518(CK-101) is an orally available third-generation and selective inhibitor of certain epidermal growth factor …

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 …

SELPERCATINIB

It’s only fair to share… SELPERCATINIB LOXO 292 CAS: 2152628-33-4 Chemical Formula: C29H31N7O3 Molecular Weight: 525.613 CEGM9YBNGD UNII-CEGM9YBNGD  6-(2-hydroxy-2-methylpropoxy)-4-(6-{6-[(6-methoxypyridin- 3-yl)methyl]-3,6-diazabicyclo[3.1.1]heptan-3-yl}pyridin-3- yl)pyrazolo[1,5-a]pyridine-3-carbonitrile Selpercatinib is a tyrosine kinase inhibitor with antineoplastic properties. A phase I/II trial is also under way in pediatric patients and young adults with activating RET alterations and advanced solid or primary CNS tumors. …