Tag «Orphan drug designation»

Caplacizumab-yhdp, カプラシズマブ

It’s only fair to share… FDA approves first therapy Cablivi (caplacizumab-yhdp) カプラシズマブ  , for the treatment of adult patients with a rare blood clotting disorder FDA February 6, 2019 Release https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm630851.htm?utm_campaign=020919_PR_FDA%20approves%20therapy%20for%20treatment%20of%20adult%20patients%20with%20rare%20blood%20clotting%20disorder&utm_medium=email&utm_source=Eloqua The U.S. Food and Drug Administration today approved Cablivi (caplacizumab-yhdp) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, …

Tagraxofusp タグラクソフスプ

It’s only fair to share… MGADDVVDSS KSFVMENFSS YHGTKPGYVD SIQKGIQKPK SGTQGNYDDD WKGFYSTDNK YDAAGYSVDN ENPLSGKAGG VVKVTYPGLT KVLALKVDNA ETIKKELGLS LTEPLMEQVG TEEFIKRFGD GASRVVLSLP FAEGSSSVEY INNWEQAKAL SVELEINFET RGKRGQDAMY EYMAQACAGN RVRRSVGSSL SCINLDWDVI RDKTKTKIES LKEHGPIKNK MSESPNKTVS EEKAKQYLEE FHQTALEHPE LSELKTVTGT NPVFAGANYA AWAVNVAQVI DSETADNLEK TTAALSILPG IGSVMGIADG AVHHNTEEIV AQSIALSSLM VAQAIPLVGE LVDIGFAAYN FVESIINLFQ VVHNSYNRPA YSPGHKTRPH MAPMTQTTSL KTSWVNCSNM IDEIITHLKQ PPLPLLDFNN LNGEDQDILM ENNLRRPNLE AFNRAVKSLQ NASAIESILK NLLPCLPLAT AAPTRHPIHI KDGDWNEFRR …

Caplacizumab, カプラシズマブ Cablivi is the first therapeutic approved in Europe, for the treatment of a rare blood-clotting disorder

It’s only fair to share… Cablivi is the first therapeutic approved in Europe, for the treatment of a rare blood-clotting disorder On September 03, 2018, the European Commission has granted marketing authorization for Cablivi™ (caplacizumab) for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. Cablivi is …

Icosapent ethyl, イコサペント酸エチル

It’s only fair to share… Icosapent ethyl 330.5042 , C22H34O2 cas 86227-47-6 / 73310-10-8 ethyl (5Z,8Z,11Z,14Z,17Z)-icosa-5,8,11,14,17-pentaenoate Ethyl eicosapentaenoic acid イコサペント酸エチル (5Z,8Z,11Z,14Z,17Z)-Eicosapetaenoic acid ethyl ester (all-Z)-5,8,11,14,17-Eicosapentaenoic acid ethyl ester 5,8,11,14,17-Eicosapentaenoic acid, ethyl ester, (5Z,8Z,11Z,14Z,17Z)- [ACD/Index Name] 5,8,11,14,17-Eicosapentaenoic acid, ethyl ester, (all-Z)- 6GC8A4PAYH 86227-47-6 [RN] all-cis-5,8,11,14,17-Eicosapentaenoic Acid Ethyl Ester Timnodonic acid ethyl ester Vascepa 5,8,11,14,17-Eicosapentaenoic acid, ethyl ester, (all-Z)- (5Z,8Z,11Z,14Z,17Z)-5,8,11,14,17-Eicosapentaenoic acid ethyl …

Patisiran

It’s only fair to share… Patisiran Sense strand: GUAACCAAGAGUAUUCCAUdTdT Anti-sense strand: AUGGAAUACUCUUGGUUACdTdT RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA (G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1), ALN-18328, 6024128  , ALN-TTR02  , GENZ-438027  , SAR-438037  , 50FKX8CB2Y (UNII code)  for RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA(G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1) Nucleic Acid Sequence Sequence Length: 42, 21, 2112 a 7 c 7 g 4 t 12 umultistranded (2); modified CAS 1420706-45-1 Treatment of Amyloidosis, …

Ivosidenib,  ивосидениб , إيفوزيدينيب , 艾伏尼布 , 

It’s only fair to share… Ivosidenib AG-120; TIBSOVO FDA approves first targeted treatment Tibsovo (ivosidenib) for patients with relapsed or refractory acute myeloid leukemia who have a certain genetic mutation The U.S. Food and Drug Administration today approved Tibsovo (ivosidenib) tablets for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) …

Tecovirimat

It’s only fair to share… FDA approves the first drug with an indication for treatment of smallpox The U.S. Food and Drug Administration today approved TPOXX (tecovirimat), the first drug with an indication for treatment of smallpox. Though the World Health Organization declared smallpox, a contagious and sometimes fatal infectious disease, eradicated in 1980, there …

RG7440, Ipatasertib, アイパタセルチブ;

It’s only fair to share…   Ipatasertib GDC-0068 , RG7440 CAS 1001264-89-6, C24H32ClN5O2, 457.9962 アイパタセルチブ; イパタセルチブ; Antineoplastic, AKT serine/threonine kinase inhibitor 2(S)-(4-Chlorophenyl)-1-[4-[7(R)-hydroxy-5(R)-methyl-6,7-dihydro-5H-cyclopenta[d]pyrimidin-4-yl]piperazin-1-yl]-3-(isopropylamino)propan-1-one (2S)-2-(4-Chlorophenyl)-1-(4-((5R,7R)-7-hydroxy-5-methyl-6,7-dihydro-5H-cyclopenta(d)pyrimidin-4-yl)piperazin-1-yl(-3-((propan-2-yl)amino)propan-1-one 1-Propanone, 2-(4-chlorophenyl)-1-(4-((5R,7R)-6,7-dihydro-7-hydroxy-5-methyl-5H-cyclopentapyrimidin-4-yl)-1-piperazinyl)-3-((1-methylethyl)amino)-,  (2S)- Ipatasertib dihydrochloride 1396257-94-5 Ipatasertib (RG7440) is an experimental cancer drug in development by Roche. It is a small molecule inhibitor of Akt. It was discovered by Array Biopharma and is currently in phase II trials for treatment of breast …

TIPIFARNIB, типифарниб , تيبيفارنيب , 替匹法尼 ,

It’s only fair to share… TIPIFARNIB R-115777, NSC-702818 Categories Antineoplastic Agents P-glycoprotein/ABCB1 Inhibitors Quinolines UNIIMAT637500A CAS number 192185-72-1 +form 192185-68-5 (racemate) 192185-69-6 (racemic; fumarate) 192185-70-9 (racemic; diHCl) (+)-(R)-6-[1-Amino-1-(4-chlorophenyl)-1-(1-methylimidazol-5-yl)methyl]-4-(3-chlorophenyl)-1-methylquinolin-2(1H)-one 2(1H)-Quinolinone, 6-[(R)-amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl]-4-(3-chlorophenyl)-1-methyl- Weight Average: 489.396 Chemical Formula C27H22Cl2N4O типифарниб [Russian] [INN] تيبيفارنيب [Arabic] [INN] 替匹法尼 [Chinese] [INN] (R)-(+)-R115777 (R)-6-(Amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl)-4-(3-chlorophenyl)-1-methyl-2(1H)-quinolinone (R)-6-(amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl)-4-(3-chlorophenyl)-1-methylquinolin-2(1H)-one 2 (1H))-Quinolinone,6-(amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl)-4-(3-chlorophenyl)-1-methyl-, 2(1H )-quinolinone Title: Tipifarnib CAS Registry Number: 192185-72-1; 192185-68-5 (unspecified stereo) CAS Name: 6-[(R)-Amino(4-chlorophenyl)(1-methyl-1H-imidazol-5-yl)methyl]-4-(3-chlorophenyl)-1-methyl-2(1H)-quinolinone Manufacturers’ Codes: R-115777 Trademarks: Zarnestra …

Fedratinib

It’s only fair to share… FEDRATINIB SAR-302503; TG-101348, 6L1XP550I6, 936091-26-8 [RN], WHO 9707 Molecular Formula: C27H36N6O3S Molecular Weight: 524.684 g/mol FLT3, JAK2 http://www.ama-assn.org//resources/doc/usan/fedratinib.pdf Fedratinib had been in phase III clincial trials by Sanofi for the treatment of myelofibrosis. However, Sanofi had discontinued this research because of the safety issues. Orphan drug designation was assigned in the U.S. and in …