Category «Orphan Drug Status»

Setmelanotide

It’s only fair to share… Setmelanotide Ac-Arg-Cys(1)-D-Ala-His-D-Phe-Arg-Trp-Cys(1)-NH2   Molecular FormulaC49H68N18O9S2 Average mass1117.309 Da N-acetyl-L-arginyl-L-cysteinyl-D-alanyl-L-histidyl-D-phenylalanyl-L-arginyl-L-tryptophyl-L-cysteinamide (2->8)-disulfide 1,2-Dithia-5,8,11,14,17,20-hexaazacyclotricosane-4-carboxamide, 22-[[(2S)-2-(acetylamino)-5-[(diaminomethylene)amino]-1-oxopentyl]amino]-10-[3-[(diaminomethylene)amino]propyl]-16-(1H-imidazol-5-ylmeth yl)-7-(1H-indol-3-ylmethyl)-19-methyl-6,9,12,15,18,21-hexaoxo-13-(phenylmethyl)-, (4R,7S,10S,13R,16S,19R,22R)- [ACD/Index Name] 10011 920014-72-8 [RN] Imcivree [Trade name] N2-acetyl-L-arginyl-L-cysteinyl-D-alanyl-L-histidyl-D-phenylalanyl-L-arginyl-Ltryptophyl- L-cysteinamide, cyclic (2-8)-disulfide N7T15V1FUY RM-493, BIM-22493 UNII-N7T15V1FUY сетмеланотид [Russian] [INN] سيتميلانوتيد [Arabic] [INN] 司美诺肽 [Chinese] [INN] (4R,7S,10S,13R,16S,19R,22R)-22-[[(2S)-2-acetamido-5-(diaminomethylideneamino)pentanoyl]amino]-13-benzyl-10-[3-(diaminomethylideneamino)propyl]-16-(1H-imidazol-5-ylmethyl)-7-(1H-indol-3-ylmethyl)-19-methyl-6,9,12,15,18,21-hexaoxo-1,2-dithia-5,8,11,14,17,20-hexazacyclotricosane-4-carboxamide FDA 11/25/2020, Imcivree, To treat obesity and the control of hunger associated with pro-opiomelanocortin deficiency, a rare disorder that causes severe obesity …

Berotralstat

It’s only fair to share… Berotralstat CAS  1809010-50-1 DIHCl 1809010-52-3 Molecular Formula, C30-H26-F4-N6-O, Molecular Weight, 562.5684 1-(3-(Aminomethyl)phenyl)-N-(5-((R)-(3-cyanophenyl)((cyclopropylmethyl)amino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide 1H-Pyrazole-5-carboxamide, 1-(3-(aminomethyl)phenyl)-N-(5-((R)-(3-cyanophenyl)((cyclopropylmethyl)amino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)- To treat patients with hereditary angioedema FDA APPROVED 12/4/2020, Orladeyo, 110MG CAPSULE 0RAL New Drug Application (NDA): 214094 Company: BIOCRYST PHARMACEUTICALS INC New Drug Application (NDA): 214094 Company: BIOCRYST PHARMACEUTICALS INC Berotralstat Hydrochloride 1-[3-(Aminomethyl)phenyl]-N-(5-{(1R)-(3-cyanophenyl)[(cyclopropylmethyl)amino]methyl}-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide dihydrochloride C30H26F4N6O▪2HCl : 635.48 [1809010-52-3] Berotralstat, also known as BCX-7353, is a kallikrein inhibitor. BCX7353 …

Pemigatinib

It’s only fair to share…   Pemigatinib INCB054828 Formula C24H27F2N5O4 CAS 1513857-77-6 2379919-96-5  HCL Mol weight 487.4991 2020/4/17FDA APPROVED, PEMAZYRE 佩米替尼 [Chinese] [INN] 3-(2,6-Difluoro-3,5-dimethoxyphenyl)-1-ethyl-8-(morpholinomethyl)-1,3,4,6-tetrahydro-2H-pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one 2H-Pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one, 3-(2,6-difluoro-3,5-dimethoxyphenyl)-1-ethyl-1,3,4,7-tetrahydro-8-(4-morpholinylmethyl)- 3-(2,6-difluoro-3,5-dimethoxyphenyl)-1-ethyl-8-(morpholin-4-ylmethyl)-1,3,4,7-tetrahydro-2H-pyrrolo[3′,2′:5,6]pyrido[4,3-d]pyrimidin-2-one OriginatorIncyte Corporation DeveloperIncyte Corporation; Innovent Biologics ClassAntineoplastics; Ethers; Fluorobenzenes; Morpholines; Pyridines; Pyrimidinones; Pyrroles; Small molecules Mechanism of ActionType 1 fibroblast growth factor receptor antagonists; Type 3 fibroblast growth factor receptor …

Bulevirtide acetate

It’s only fair to share…   Bulevirtide acetate (N-Myristoyl-glycyl-L-threonyl-L-asparaginyl-L-leucyl-L-seryl-L-valyl-Lprolyl-L-asparaginyl-L-prolyl-L-leucyl-glycyl-L-phenylalanyl-L-phenylalanyl-L-prolyl-L-aspartyl-L-histidyl-Lglutaminyl-L-leucyl-L-aspartyl-L-prolyl-L-alanyl-L-phenylalanyl-glycyl-L-alanyl-L-asparaginyl-L-seryl-Lasparaginyl-L-asparaginyl-Lprolyl-L-aspartyl-L-tryptophanyl-L-aspartyl-L-phenylalanyl-L-asparaginyl-L-prolylL-asparaginyl-L-lysyl-L-aspartyl-L-histidyl-L-tryptophanyl-L-prolyl-L-glutamyl-L-alanyl-L-asparaginyl-L-lysylL-valylglycinamide, acetate salt. molecular formula C248H355N65O72, molecular mass is 5398.9 g/mol ブレビルチド酢酸塩; APROVED 2020/7/31, EU, Hepcludex MYR GmbH Antiviral, Entry inhibitor   Disease Hepatitis delta virus infection Bulevirtide is a 47-amino acid peptide with a fatty acid, a myristoyl residue, at the N-terminus and an amidated C-terminus. The active substance …

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; …