Category «Priority review»

First FDA-approved vaccine Dengvaxia for the prevention of dengue disease in endemic regions

First FDA-approved vaccine for the prevention of dengue disease in endemic regions May 01, 2019 The U.S. Food and Drug Administration announced today the approval of Dengvaxia, the first vaccine approved for the prevention of dengue disease caused by all dengue virus serotypes (1, 2, 3 and 4) in people ages 9 through 16 who …

FDA approves first treatment Benlysta (belimumab) for pediatric patients with lupus

  The U.S. Food and Drug Administration today approved Benlysta (belimumab) intravenous (IV) infusion for treatment of children with systemic lupus erythematosus (SLE) – often referred to as simply “lupus” – a serious chronic disease that causes inflammation and damage to various body tissues and organs. This is the first time that the FDA has …

ブレキサノロン , Brexanolone, Allopregnanolone

  Brexanolone 318.501 g/mol, C21H34O2 CAS: 516-54-1 ブレキサノロン MFCD00003677 Pregnan-20-one, 3-hydroxy-, (3α,5α)- Pregnan-20-one, 3-hydroxy-, (3α,5α)- [ACD/Index Name] S39XZ5QV8Y TU4383000 UNII:S39XZ5QV8Y (1S,2S,7S,11S,14S,15S,5R,10R)-14-acetyl-5-hydroxy-2,15-dimethyltetracyclo[8.7.0.0<2,7>.0<11,15>]heptadecane (+)-3a-Hydroxy-5a-pregnan-20-one (+)-3α-Hydroxy-5α-pregnan-20-one (3α,5α)-3-Hydroxypregnan-20-one [ACD/IUPAC Name] 10446 3211363 [Beilstein] 3a-Hydroxy-5a-pregnan-20-one The U.S. Food and Drug Administration today approved Zulresso (brexanolone) injection for intravenous (IV) use for the treatment of postpartum depression (PPD) in adult women. This is the first drug …

Caplacizumab-yhdp, カプラシズマブ

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, for the treatment of adult …

Tagraxofusp タグラクソフスプ

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 KLTFYLKTLE NAQAQQTTLS LAIF (disulfide bridge: …

USFDA approval to Lumoxiti (moxetumomab pasudotoxtdfk) a new treatment for hairy cell leukemia

USFDA approval to Lumoxiti is a new treatment for hairy cell leukemia On September 13, 2018, the U.S. Food and Drug Administration approved Lumoxiti (moxetumomab pasudotoxtdfk) injection for intravenous use for the treatment of adult patients with relapsed or refractory Hairy Cell Leukemia (HCL) who have received at least two prior systemic therapies, including treatment …

FDA approves first drug Oxervate (cenegermin) for neurotrophic keratitis, a rare eye disease

http://www.who.int/medicines/publications/druginformation/issues/77_INN_Recommended_List.pdf Active Substance General information The active substance in Oxervate, cenegermin, is a recombinant human Nerve Growth factor (rhNGF) produced in E. coli strain HMS174. The molecule is identical to human Nerve Growth factor (NGF), a naturally occurring human protein. In humans, NGF is naturally produced as pre-pro-peptide, secreted into the endoplasmic reticulum and cleaved …

Patisiran

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, SEE…..https://endpts.com/gung-ho-alnylam-lands-historic-fda-ok-on-patisiran-revving-up-the-first-global-rollout-for-an-rnai-breakthrough/ Lipid-nanoparticle-encapsulated double-stranded siRNA targeting …

FDA approves first treatment Azedra (iobenguane I 131) for rare adrenal tumors

  Iobenguane I 131   FDA approves first treatment for rare adrenal tumors The U.S. Food and Drug Administration today approved Azedra (iobenguane I 131) injection for intravenous use for the treatment of adults and adolescents age 12 and older with rare tumors of the adrenal gland (pheochromocytoma or paraganglioma) that cannot be surgically removed …

TAFENOQUINE, タフェノキン

Tafenoquine タフェノキン N-[2,6-dimethoxy-4-methyl-5-[3-(trifluoromethyl)phenoxy]quinolin-8-yl]pentane-1,4-diamine 1,4-Pentanediamine, N4-[2,6-dimethoxy-4-methyl-5-[3-(trifluoromethyl)phenoxy]-8-quinolinyl]- 106635-80-7 [RN] 262P8GS9L9 7835 N4-{2,6-Dimethoxy-4-methyl-5-[3-(trifluormethyl)phenoxy]-8-chinolinyl}-1,4-pentandiamin WR-238605, WR 238605, cas no 106635-80-7, Tafenoquine succinate, Etaquine, SB-252263, WR-238605 N(4)-(2,6-Dimethoxy-4-methyl-5-((3-trifluoromethyl)phenoxy)-8-quinolinyl)-1,4-pentanediamine Molecular Formula: C24H28F3N3O3 Molecular Weight: 463.49263 Medicines for Malaria Venture Walter Reed Army Institute (Originator) PATENT  US 4617394 Synonyms Etaquine[5] WR 238605 [5] SB-252263 New Drug Application (NDA): 210795 Company: GLAXOSMITHKLINE FDA approved on July 20, 2018 FDA  NDA 210795 Orphan This new …