Category «FDA 2023»

Perfluorhexyloctane

It’s only fair to share… WeightAverage: 432.269Monoisotopic: 432.112266666Chemical FormulaC14H17F13 Perfluorhexyloctane 1,1,1,2,2,3,3,4,4,5,5,6,6-tridecafluorotetradecane FDA APPROVED 8/16/2023, Sohonos, To reduce the volume of new heterotopic ossification in adults and pediatric patients (aged 8 years and older for females and 10 years and older for males) with fibrodysplasia ossificans progressivaDrug Trials Snapshot Perfluorohexyloctane is a fluoroalkane that is tetradecane in which …

Tofersen

It’s only fair to share… all-P-ambo-2′-O-(2-Methoxyethyl)-5-methyl-P-thiocytidylyl-(3’→5′)-2′-O-(2-methoxyethyl)adenylyl-(3’→5′)-2′-O-(2-methoxyethyl)-P-thioguanylyl-(3’→5′)-2′-O-(2-methoxyethyl)guanylyl-(3’→5′)-2′-O-(2-methoxyethyl)-P-thioadenylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-2′-deoxy-P-thioadenylyl-(3’→5′)-2′-deoxy-5-methyl-P-thiocytidylyl-(3’→5′)-2′-deoxy-P-thioadenylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-2′-deoxy-5-methyl-P-thiocytidylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-2′-deoxy-P-thioadenylyl-(3’→5′)-2′-O-(2-methoxyethyl)-5-methylcytidylyl-(3’→5′)-2′-O-(2-methoxyethyl)-P-thioadenylyl-(3’→5′)-2′-O-(2-methoxyethyl)guanylyl-(3’→5′)-2′-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3’→5′)-2′-O-(2-methoxyethyl)-5-methyluridine C230H317N72O123P19S15  : 7127.86[2088232-70-4] Tofersen CAS 2088232-70-4 FDA APPROVED 4/25/2023, Qalsody To treat amyotrophic lateral sclerosis in adults who have a SOD1 gene mutationDrug Trials Snapshot A nucleic acid-based drug indicated for the treatment of a specific type of amyotrophic lateral sclerosis. Tofersen, sold under the brand name Qalsody, is a medication used for the …

Leniolisib

It’s only fair to share… Leniolisib CAS 1354690-24-6 WeightAverage: 450.466Monoisotopic: 450.199108558 Chemical FormulaC21H25F3N6O2 1-[(3S)-3-({6-[6-methoxy-5-(trifluoromethyl)pyridin-3-yl]-5H,6H,7H,8H-pyrido[4,3-d]pyrimidin-4-yl}amino)pyrrolidin-1-yl]propan-1-one FDA APPROVED Joenja, 3/24/2023, To treat activated phosphoinositide 3-kinase delta syndromeDrug Trials Snapshot Leniolisib (INN[3][4]), sold under the brand name Joenja, is a medication used for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS).[2][5] It is a kinase inhibitor[2][6] that is taken by mouth.[2] The most common side effects …

Rezafungin

It’s only fair to share… Rezafungin CAS 1396640-59-7 WeightAverage: 1226.411Monoisotopic: 1225.602719729 Chemical FormulaC63H85N8O17 FDA APPROVED 3/22/2023, Rezzayo, To treat candidemia and invasive candidiasisDrug Trials Snapshot 2-[[(3S,6S,9S,11R,15S,18S,20R,21R,24S,25S,26S)-6-[(1S,2S)-1,2-dihydroxy-2-(4-hydroxyphenyl)ethyl]-11,20,25-trihydroxy-3,15-bis[(1R)-1-hydroxyethyl]-26-methyl-2,5,8,14,17,23-hexaoxo-18-[[4-[4-(4-pentoxyphenyl)phenyl]benzoyl]amino]-1,4,7,13,16,22-hexazatricyclo[22.3.0.09,13]heptacosan-21-yl]oxy]ethyl-trimethylazanium Rezafungin, sold under the brand name Rezzayo (by Melinta Therapeutics), is a medication used for the treatment of invasive candidiasis.[2] It is an echinocandin antifungal[1][4] that acts as a fungal β-glucan synthase inhibitor.[5] Rezafungin was approved for medical use in …

Omaveloxolone

It’s only fair to share… Omaveloxolone CAS 1474034-05-3 N-[(4aS,6aR,6bS,8aR,12aS,14aR,14bS)-11-cyano-2,2,6a,6b,9,9,12a-heptamethyl-10,14-dioxo-1,2,3,4,4a,5,6,6a,6b,7,8,8a,9,10,12a,14,14a,14b-octadecahydropicen-4a-yl]-2,2-difluoropropanamide N-[(4aS,6aR,6bS,8aR,12aS,14aR,14bS)-11-cyano-2,2,6a,6b,9,9,12a-heptamethyl-10,14-dioxo-1,3,4,5,6,7,8,8a,14a,14b-decahydropicen-4a-yl]-2,2-difluoropropanamide FDA 2023, 2/28/2023, To treat Friedrich’s ataxiaDrug Trials Snapshot WeightAverage: 554.723Monoisotopic: 554.331999611 Chemical FormulaC33H44F2N2O3 Omaveloxolone, sold under the brand name Skyclarys, is a medication used for the treatment of Friedreich’s ataxia.[2][5] It is taken by mouth.[2] The most common side effects include an increase in alanine transaminase and an increase of …

Elacestrant 

It’s only fair to share… Elacestrant  (6R)-6-[2-[ethyl-[[4-[2-(ethylamino)ethyl]phenyl]methyl]amino]-4-methoxyphenyl]-5,6,7,8-tetrahydronaphthalen-2-ol (6R)-6-{2-[ethyl({4-[2-(ethylamino)ethyl]phenyl}methyl)amino]-4-methoxyphenyl}-5,6,7,8-tetrahydronaphthalen-2-ol FDA 1/27/2023, Orserdu WeightAverage: 458.646Monoisotopic: 458.293328472 Chemical FormulaC30H38N2O2 To treat estrogen receptor-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated, advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapyDrug Trials Snapshot Elacestrant, sold under the brand name Orserdu, is a selective estrogen receptor …

Pirtobrutinib

It’s only fair to share… Pirtobrutinib 5-amino-3-[4-[[(5-fluoro-2-methoxybenzoyl)amino]methyl]phenyl]-1-[(2S)-1,1,1-trifluoropropan-2-yl]pyrazole-4-carboxamide FDA 2023, 1/27/2023, Jaypirca To treat relapsed or refractory mantle cell lymphoma in adults who have had at least two lines of systemic therapy, including a BTK inhibitorDrug Trials Snapshot Pirtobrutinib, sold under the brand name Jaypirca, is an anticancer medication that is used to treat mantle cell lymphoma.[1][2][4] It inhibits B cell …

Capivasertib

It’s only fair to share… Capivasertib C21H25ClN6O2 428.915 1143532-39-1 AZD 5363 4-amino-N-[(1S)-1-(4-chlorophenyl)-3-hydroxypropyl]-1-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)piperidine-4-carboxamide (S)-4-AMINO-N-(1-(4-CHLOROPHENYL)-3-HYDROXYPROPYL)-1-(7H-PYRROLO[2,3-D]PYRIMIDIN-4-YL)PIPERIDINE-4-CARBOXAMIDE FDA APPROVED 11/16/2023, To treat breast cancer that meets certain disease criteria, Truqap Capivasertib, sold under the brand name Truqap, is an anti-cancer medication used for the treatment of breast cancer.[1][2] The most common adverse reactions include diarrhea, cutaneous adverse reactions, increased random glucose, decreased lymphocytes, decreased hemoglobin, increased …

Eplontersen

It’s only fair to share…     Eplontersen AKCEA-TTR-LRx   ION-682884 FREE ACID ISIS-682884 FREE ACID UNII0GRZ0F5XJ6 CAS number1637600-16-8 ALL-P-AMBO-5-O-(((6-(5-((TRIS(3-(6-(2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYLOXY)HEXYLAMINO)-3-OXOPROPOXYMETHYL))METHYL)AMINO-5-OXOPENTANAMIDO)HEXOXY))PHOSPHO)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)GUANYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-2-DEOXY-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)ADENYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTIDINE FDA APP, 12/21/2023, To treat polyneuropathy of hereditary transthyretin-mediated amyloidosis, Wainua AKCEA-TTR-LRx is under investigation in clinical trial NCT04136184 (Neuro-ttransform: A Study to Evaluate the Efficacy and Safety of Akcea-ttr-lrx in Participants With Hereditary Transthyretin-mediated Amyloid Polyneuropathy). Eplontersen, …

Iptacopan

It’s only fair to share… Iptacopan 1644670-37-0 422.525, C25H30N2O4 4-((2S,4S)-4-ethoxy-1-((5-methoxy-7-methyl-1H-indol-4-yl)methyl)piperidin-2-yl) benzoic acid BENZOIC ACID, 4-((2S,4S)-4-ETHOXY-1-((5-METHOXY-7-METHYL-1H-INDOL-4-YL)METHYL)-2-PIPERIDINYL)- Iptacopan   LNP 023 LNP-023 LNP023 NVP-LNP023 NVP-LNP023-NX   fda approved, To treat paroxysmal nocturnal hemoglobinuria, 12/5/2023, Fabhalta ‘ Iptacopan is a small-molecule factor B inhibitor previously investigated as a potential treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) by inhibiting the …