Category «orphan drug»

Pracinostat

It’s only fair to share… Pracinostat Molecular Formula C20H30N4O2 Average mass 358.478 Da 2-Propenamide, 3-[2-butyl-1-[2-(diethylamino)ethyl]-1H-benzimidazol-5-yl]-N-hydroxy-, (2E)- 929016-96-6 [RN] SB939 (2E)-3-{2-butyl-1-[2-(diethylamino)ethyl]-1,3-benzodiazol-5-yl}-N-hydroxyprop-2-enamide N-hydroxy-1-[(4-methoxyphenyl)methyl]-1H-indole-6-carboxamide PCI 34051,  UNII: GPO2JN4UON 929016-98-8 DI HCl salt, C20 H30 N4 O2 . 2 Cl H, 431.4 929016-96-6 (free base) 929016-97-7 (trifluoroacetate) S*BIO (Originator) Leukemia, acute myeloid, phase 3, helsinn CAS 929016-98-8 DI HCl salt, C20 H30 N4 O2 . 2 Cl …

BMS-986020

It’s only fair to share… BMS-986020 AM-152; BMS-986020; BMS-986202 cas 1257213-50-5 Chemical Formula: C29H26N2O5 Molecular Weight: 482.536 (R)-1-(4′-(3-methyl-4-(((1-phenylethoxy)carbonyl)amino)isoxazol-5-yl)-[1,1′-biphenyl]-4-yl)cyclopropane-1-carboxylic acid Cyclopropanecarboxylic acid, 1-(4′-(3-methyl-4-((((1R)-1-phenylethoxy)carbonyl)amino)-5-isoxazolyl)(1,1′-biphenyl)-4-yl)- 1-(4′-(3-Methyl-4-(((((R)-1-phenylethyl)oxy)carbonyl)amino)isoxazol-5-yl)biphenyl-4-yl)cyclopropanecarboxylic acid UNII: 38CTP01B4L For treatment for pulmonary fibrosis, phase 2, The lysophosphatidic acid receptor, LPA1, has been implicated as a therapeutic target for fibrotic disorders Lysophospholipids (LPs), including lysophosphatidic acid (LPA), sphingosine 1-phospate (S1P), …

FDA approves first subcutaneous C1 Esterase Inhibitor to treat rare genetic disease

It’s only fair to share… FDA approves first subcutaneous C1 Esterase Inhibitor to treat rare genetic disease 06/22/2017   The U.S. Food and Drug Administration today approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous (under the skin) administration to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients. The subcutaneous route of …

FDA approves drug to treat ALS, Radicava (Edaravone) , эдаравон, إيدارافون , 依达拉奉 ,ラジカット,

It’s only fair to share… FDA approves drug to treat ALS 05/05/2017 The U.S. Food and Drug Administration today approved Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease. May 5, 2017 Release The U.S. Food and Drug Administration today approved Radicava (edaravone) to treat patients with …

FDA approves new combination treatment for acute myeloid leukemia, Rydapt (midostaurin)

It’s only fair to share… MIDOSTAURIN FDA approves new combination treatment for acute myeloid leukemia 04/28/2017 The U.S. Food and Drug Administration today approved Rydapt (midostaurin) for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who have a specific genetic mutation called FLT3, in combination with chemotherapy. The drug is approved …

FDA approves first treatment for a form of Batten disease, Brineura (cerliponase alfa)

It’s only fair to share… FDA approves first treatment for a form of Batten disease 04/27/2017 The U.S. Food and Drug Administration today approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease. Brineura is the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic pediatric patients 3 …

BLU 285

It’s only fair to share… BLU-285 CAS 1703793-34-3 Molecular FormulaC26H27FN10 Average mass498.558 Da (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 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)- 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)- Originator Blueprint Medicines Class Antineoplastics; Skin disorder therapies; Small molecules Mechanism of Action Platelet-derived growth factor alpha receptor modulators; Proto oncogene protein c-kit inhibitors Orphan Drug Status Yes – Systemic mastocytosis; Gastrointestinal stromal tumours Phase I Gastrointestinal stromal tumours; Solid tumours; …

TRIENTINE HYDROCHLORIDE, 塩酸トリエンチン , 曲恩汀

It’s only fair to share… TRIENTINE Molecular Formula C6H18N4 Average mass 146.234 Da 112-24-3 CAS 曲恩汀, KD-034, MK-0681, MK-681, TECZA, TETA, TJA-250 1,2-Ethanediamine, N1,N2-bis(2-aminoethyl)- 1,8-diamino-3,6-diazaoctane TRIENTINE HYDROCHLORIDE Molecular Formula C6H19ClN4 Average mass 182.695 Da 38260-01-4 CAS Launched – 1986 VALEANT, WILSONS DISEASE 塩酸トリエンチン Trientine Hydrochloride C6H18N4▪2HCl : 219.16 [38260-01-4] Aton Pharma, a subsidiary of Valeant Pharmaceuticals, has developed and launched Syprine, a capsule formulation of trientine hydrochloride, …

FDA approves Xermelo (telotristat ethyl) for carcinoid syndrome diarrhea

It’s only fair to share…   Telotristat ethyl Molecular Formula, C27-H26-Cl-F3-N6-O3, Molecular Weight, 574.9884, RN: 1033805-22-9 UNII: 8G388563M LX 1032 (2S)-2-Amino-3-[4-[2-amino-6-[[(1R)-1-[4-chloro-2-(3-methylpyrazol-1-yl)phenyl]-2,2,2-trifluoroethyl]oxy]pyrimidin-4-yl]phenyl]propionic acid ethyl ester Ethyl-4-(2-amino-6-{(1R)-1-[4-chlor-2-(3-methyl-1H-pyrazol-1-yl)phenyl]-2,2,2-trifluorethoxy}-4-pyrimidinyl)-L-phenylalaninat L-Phenylalanine, 4-[2-amino-6-[(1R)-1-[4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl]-2,2,2-trifluoroethoxy]-4-pyrimidinyl]-, ethyl ester SEE…………… Telotristat etiprate, (S)-ethyl 2-amino-3-(4-(2-amino-6-((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate 2-benzamidoacetate . CAS: 1137608-69-5 (etiprate), LX 1606 Chemical Formula: C36H35ClF3N7O6 Molecular Weight: 754.16 L-Phenylalanine, 4-[2-amino-6-[(1R)-1-[4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl]-2,2,2-trifluoroethoxy]-4-pyrimidinyl]-, ethyl ester, compd. with N-benzoylglycine (1:1) LX 1032 …

Deflazacort

It’s only fair to share…   Deflazacort CAS 14484-47-0 Molecular Formula C25H31NO6 Average mass 441.517 Da (11b,16b)-21-(Acetyloxy)-11-hydroxy-2′-methyl-5’H-pregna-1,4-dieno[17,16-d]oxazole-3,20-dione 11b,21-Dihydroxy-2′-methyl-5’bH-pregna-1,4-dieno[17,16-d]oxazole-3,20-dione 21-acetate 2-[(4aR,4bS,5S,6aS,6bS,9aR,10aS,10bS)-5-Hydroxy-4a,6a,8-trimethyl-2-oxo-2,4a,4b,5,6,6a,9a,10,10a,10b,11,12-dodecahydro-6bH-naphtho[2′,1′:4,5]indeno[1,2-d][1,3]oxazol-6b-yl]-2-oxoethyl acetate 5’βH-Pregna-1,4-dieno[17,16-d]oxazole-3,20-dione, 11β,21-dihydroxy-2′-methyl-, 21-acetate (8CI) (11β,16β)-21-(Acetyloxy)-11-hydroxy-2′-methyl-5’H-pregna-1,4-dieno[17,16-d]oxazole-3,20-dione 2H-Naphth[2′,1′:4,5]indeno[1,2-d]oxazole, 5’H-pregna-1,4-dieno[17,16-d]oxazole-3,20-dione deriv. Azacort Azacortinol Calcort DL 458IT Deflan Optical Rotatory Power +62.3 ° Conc: 0.5 g/100mL; Solv: chloroform (67-66-3); Wavlength: 589.3 nm …………..REF, “Drugs – Synonyms and Properties” data were obtained …