Category «Orphan Drug Status»

Teprotumumab-trbw

It’s only fair to share… Tepezza (teprotumumab-trbw) Company: Horizon Therapeutics plc Date of Approval: January 21, 2020 Treatment for: Thyroid Eye Disease Labels for BLA 761143 UNIIY64GQ0KC0A CAS number1036734-93-6 R-1507 / R1507 / RG-1507 / RG1507 / RO-4858696 / RO-4858696-000 / RO-4858696000 / RO4858696 / RO4858696-000 / RV-001 / RV001 Tepezza (teprotumumab-trbw) is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor 1 receptor (IGF-1R) for the treatment of active thyroid eye …

Brilliant blue G , ブリリアントブルーG ,

It’s only fair to share…   Brilliant blue G FDA 2019, 12/20/2019, TISSUEBLUE, New Drug Application (NDA): 209569 Company: DUTCH OPHTHALMIC, PRIORITY; Orphan OPQ recommends APPROVAL of NDA 209569 for commercialization of TissueBlue (Brilliant Blue G Ophthalmic Solution), 0.025% Neuroprotectant sodium;3-[[4-[[4-(4-ethoxyanilino)phenyl]-[4-[ethyl-[(3-sulfonatophenyl)methyl]azaniumylidene]-2-methylcyclohexa-2,5-dien-1-ylidene]methyl]-N-ethyl-3-methylanilino]methyl]benzenesulfonate Formula C47H48N3O7S2. Na CAS 6104-58-1 Mol weight 854.0197 ブリリアントブルーG, C.I. Acid Blue 90 UNII-M1ZRX790SI M1ZRX790SI 6104-58-1 Brilliant Blue G Derma …

Avapritinib, アバプリチニブ , авапритиниб , أفابريتينيب ,

It’s only fair to share… Avapritinib BLU-285, BLU285 Antineoplastic, Tyrosine kinase inhibitor アバプリチニブ авапритиниб [Russian] [INN] أفابريتينيب [Arabic] [INN] (1S)-1-(4-fluorophenyl)-1-[2-[4-[6-(1-methylpyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]piperazin-1-yl]pyrimidin-5-yl]ethanamine (1S)-1-(4-Fluorophenyl)-1-(2-{4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl}-5-pyrimidinyl)ethanamine 10613 1703793-34-3 [RN] 513P80B4YJ 5-Pyrimidinemethanamine, α-(4-fluorophenyl)-α-methyl-2-[4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl]-, (αS)- (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-1-yl)pyrimidin-5-yl)ethan-1-amine (αS)-(4-fluorophenyl)-α-methyl-2-[4-[6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]-1-piperazinyl]-5-pyrimidinemethanamine Formula C26H27FN10 CAS 1703793-34-3 Mol weight 498.558 No. Drug Name Active Ingredient Approval Date FDA-approved use on approval date* 1. Ayvakit avapritinib 1/9/2020 To treat adults with unresectable or metastatic …

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 …

FDA approves first treatment Givlaari (givosiran) for inherited rare disease

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 …

Blarcamesine, ブラルカメシン ,

It’s only fair to share… Blarcamesine ブラルカメシン; [(2,2-diphenyloxolan-3-yl)methyl]dimethylamine Anavex 2-73 Tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanemethanamine THD-DP-FM AE-37 / AE37 / ANAVEX 2-73 FREE BASE UNII 9T210MMZ3F Formula C19H23NO Cas 195615-83-9 195615-84-0 HCL Mol weight 281.392 Treatment of Rett syndrome, Investigated for use/treatment in breast cancer. Anti-amnesic, Muscarinic/sigma receptor agonist Originator Anavex Life Sciences Developer ABX-CRO; Anavex Life Sciences; The Michael J. Fox Foundation for Parkinsons Research Class Antidementias; …

FDA approves first treatment Soliris (eculizumab) for neuromyelitis optica spectrum disorder, a rare autoimmune disease of the central nervous system

It’s only fair to share… The U.S. Food and Drug Administration today approved Soliris (eculizumab) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. NMOSD is an autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal …

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. …