It’s only fair to share… (Heavy chain) QVQLVQSGAE VKKPGASVKV SCKASGYSFT SSWIHWVKQA PGQGLEWMGE INPGNVRTNY NENFRNKVTM TVDTSISTAY MELSRLRSDD TAVYYCTVVF YGEPYFPYWG QGTLVTVSSA STKGPSVFPL APSSKSTSGG TAALGCLVKD YFPEPVTVSW NSGALTSGVH TFPAVLQSSG LYSLSSVVTV PSSSLGTQTY ICNVNHKPSN TKVDKRVEPK SCDKTHTCPP CPAPEAAGGP SVFLFPPKPK DTLMISRTPE VTCVVVDVSH EDPEVKFNWY VDGVEVHNAK TKPREEQYNS TYRVVSVLTV LHQDWLNGKE YKCKVSNKAL PAPIEKTISK AKGQPREPQV YTLPPSREEM TKNQVSLTCL VKGFYPSDIA VEWESNGQPE NNYKTTPPVL DSDGSFFLYS KLTVDKSRWQ QGNVFSCSVM HEALHNHYTQ KSLSLSPGK (Light chain) QIVLTQSPGT …
Tag «Orphan Drug Status»
MIRDAMETINIB
It’s only fair to share… MIRDAMETINIB 391210-10-9 Chemical Formula: C16H14F3IN2O4 Molecular Weight: 482.19 PD0325901; PD 0325901; PD-325901; mirdametinib IUPAC/Chemical Name: (R)-N-(2,3-dihydroxypropoxy)-3,4-difluoro-2-((2-fluoro-4-iodophenyl)amino)benzamide SpringWorks Therapeutics (a spin out of Pfizer ) is developing mirdametinib, a second-generation, non-ATP competitive, allosteric MEK1 and MEK2 inhibitor derived from CI-1040, for treating type 1 neurofibromatosis (NF1) and advanced solid tumors. In June …
Dasiglucagon
It’s only fair to share… Dasiglucagon Treatment of Hypoglycemia in Type 1 and Type 2 Diabetes Patients Formula C152H222N38O50 CAS 1544300-84-6 Mol weight 3381.6137 FDA APPROVED, 2021/3/22, Zegalogue Zealand Pharma A/S UNIIAD4J2O47FQ HypoPal rescue pen SVG Image IUPAC Condensed H-His-Ser-Gln-Gly-Thr-Phe-Thr-Ser-Asp-Tyr-Ser-Lys-Tyr-Leu-Asp-Aib-Ala-Arg-Ala-Glu-Glu-Phe-Val-Lys-Trp-Leu-Glu-Ser-Thr-OH Sequence HSQGTFTSDYSKYLDXARAEEFVKWLEST HELM PEPTIDE1{H.S.Q.G.T.F.T.S.D.Y.S.K.Y.L.D.[Aib].A.R.A.E.E.F.V.K.W.L.E.S.T}$$$$ IUPAC L-histidyl-L-seryl-L-glutaminyl-glycyl-L-threonyl-L-phenylalanyl-L-threonyl-L-seryl-L-alpha-aspartyl-L-tyrosyl-L-seryl-L-lysyl-L-tyrosyl-L-leucyl-L-alpha-aspartyl-alpha-methyl-alanyl-L-alanyl-L-arginyl-L-alanyl-L-alpha-glutamyl-L-alpha-glutamyl-L-phenylalanyl-L-valyl-L-lysyl-L-tryptophyl-L-leucyl-L-alpha-glutamyl-L-seryl-L-threonine (4S)-4-[[(2S)-2-[[(2S)-2-[[(2S)-2-[[2-[[(2S)-2-[[(2S)-2-[[(2S)-2-[[(2S)-6-amino-2-[[(2S)-2-[[(2S)-2-[[(2S)-2-[[(2S)-2-[[(2S,3R)-2-[[(2S)-2-[[(2S,3R)-2-[[2-[[(2S)-5-amino-2-[[(2S)-2-[[(2S)-2-amino-3-(1H-imidazol-4-yl)propanoyl]amino]-3-hydroxypropanoyl]amino]-5-oxopentanoyl]amino]acetyl]amino]-3-hydroxybutanoyl]amino]-3-phenylpropanoyl]amino]-3-hydroxybutanoyl]amino]-3-hydroxypropanoyl]amino]-3-carboxypropanoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-3-hydroxypropanoyl]amino]hexanoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]-4-methylpentanoyl]amino]-3-carboxypropanoyl]amino]-2-methylpropanoyl]amino]propanoyl]amino]-5-carbamimidamidopentanoyl]amino]propanoyl]amino]-5-[[(2S)-1-[[(2S)-1-[[(2S)-1-[[(2S)-6-amino-1-[[(2S)-1-[[(2S)-1-[[(2S)-4-carboxy-1-[[(2S)-1-[[(1S,2R)-1-carboxy-2-hydroxypropyl]amino]-3-hydroxy-1-oxopropan-2-yl]amino]-1-oxobutan-2-yl]amino]-4-methyl-1-oxopentan-2-yl]amino]-3-(1H-indol-3-yl)-1-oxopropan-2-yl]amino]-1-oxohexan-2-yl]amino]-3-methyl-1-oxobutan-2-yl]amino]-1-oxo-3-phenylpropan-2-yl]amino]-4-carboxy-1-oxobutan-2-yl]amino]-5-oxopentanoic acid . [16-(2-methylalanine)(S>X),17-L-alanine(R>A),20-L-α-glutamyl(Q>E),21-L-αglutamyl(D>E),24-L-lysyl(Q>K),27-L-α-glutamyl(M>E),28-L-serine(N>S)]human glucagon L-Threonine, L-histidyl-L-seryl-L-glutaminylglycyl-L-threonyl-L- …
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 …
Odevixibat
It’s only fair to share… Odevixibat A-4250, AR-H 064974 CAS 501692-44-0 BUTANOIC ACID, 2-(((2R)-2-((2-((3,3-DIBUTYL-2,3,4,5-TETRAHYDRO-7-(METHYLTHIO)-1,1-DIOXIDO-5-PHENYL-1,2,5-BENZOTHIADIAZEPIN-8-YL)OXY)ACETYL)AMINO)-2-(4-HYDROXYPHENYL)ACETYL)AMINO)-, (2S)- (2S)-2-[[(2R)-2-[[2-[(3,3-dibutyl-7-methylsulfanyl-1,1-dioxo-5-phenyl-2,4-dihydro-1λ6,2,5-benzothiadiazepin-8-yl)oxy]acetyl]amino]-2-(4-hydroxyphenyl)acetyl]amino]butanoic acid Molecular Formula C37H48N4O8S2 Molecular Weight 740.929 AZD8294WHO 10706AR-H064974HY-109120CS-0078340D11716US9694018, 5Originator Albireo AB Developer Albireo AB; Albireo Pharma ClassAcetamides; Butyric acids; Hepatoprotectants; Small molecules; Sulfones; Thiazepines Mechanism of Action Sodium-bile acid cotransporter inhibitors Orphan Drug Status Yes – Primary biliary cirrhosis; Biliary atresia; Intrahepatic cholestasis; Alagille …
TILDACERFONT
It’s only fair to share… TILDACERFONT Synonyms: Tildacerfont 1014983-00-6 3-(4-Chloro-2-morpholin-4-yl-thiazol-5-yl)-7-(1-ethyl-propyl)-2,5-dimethyl-pyrazolo[1,5-a]pyrimidine 7-(1-ethyl-propyl)-3-(4-chloro-2-morpholin-4-yl-thiazol-5-yl)-2,5-dimethyl-pyrazolo[1,5-a]pyrimidine MW/ MF 420 g/mol/ C20H26ClN5OS Originator Spruce Biosciences Class2 ring heterocyclic compounds; Morpholines; Pyrazoles; Pyrimidines; Small molecules; Thiazoles Mechanism of Action Corticotropin receptor antagonists Orphan Drug Status Yes – Congenital adrenal hyperplasia New Molecular Entity Yes Phase II Congenital adrenal hyperplasia 09 Jul 2020 Spruce Biosciences initiates a phase II trial …
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; …
Reldesemtiv
It’s only fair to share… Reldesemtiv CK-2127107 CAS 1345410-31-2 UNII-4S0HBYW6QE, 4S0HBYW6QE MW 384.4 g/mol, MF C19H18F2N6O 1-[2-({[trans-3-fluoro-1-(3-fluoropyridin-2-yl)cyclobutyl]methyl}amino)pyrimidin-5-yl]-1H-pyrrole-3- carboxamide 1-[2-[[3-fluoro-1-(3-fluoropyridin-2-yl)cyclobutyl]methylamino]pyrimidin-5-yl]pyrrole-3-carboxamide Reldesemtiv, also known as CK-2127107, is a skeletal muscle troponin activator (FSTA) and is a potential treatment for people living with debilitating diseases and conditions associated with neuromuscular or non-neuromuscular dysfunction, muscular weakness, and/or muscle fatigue such as SMA, …
MITAPIVAT
It’s only fair to share… MITAPIVAT CAS 1260075-17-9 MF C24H26N4O3S MW 450.55 UPDATE…. FDA APPROVE 2/17/2022 To treat hemolytic anemia in pyruvate kinase deficiency, Pyrukynd митапиват [Russian] [INN] ميتابيفات [Arabic] [INN] 米他匹伐 [Chinese] [INN] 10226 1260075-17-9 [RN] 2WTV10SIKH AG-348 8-Quinolinesulfonamide, N-[4-[[4-(cyclopropylmethyl)-1-piperazinyl]carbonyl]phenyl]- N-[4-[[4-(Cyclopropylmethyl)-1-piperazinyl]carbonyl]phenyl]-8-quinolinesulfonamide Originator Agios Pharmaceuticals Class Antianaemics; Piperazines; Quinolines; Small molecules; Sulfonamides Mechanism of Action Pyruvate kinase stimulants Orphan Drug Status Yes – Inborn error metabolic …
Cavosonstat (N-91115)
It’s only fair to share… Cavosonstat (N-91115) CAS 1371587-51-7 C16H10ClNO3, 299.71 g/mol UNII-O2Z8Q22ZE4, O2Z8Q22ZE4, NCT02589236; N91115-2CF-05; SNO-6 3-chloro-4-(6-hydroxyquinolin-2-yl)benzoic acid Treatment of Chronic Obstructive Pulmonary Diseases (COPD), AND Cystic fibrosis, Nivalis Therapeutics, phase 2 The product was originated at Nivalis Therapeutics, which was acquired by Alpine Immune Sciences in 2017. In 2018, Alpine announced the sale and transfer of global rights to …