Tag «Orphan drug designation»

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 …

CK-101

It’s only fair to share…   CK-101, RX-518 CAS 1660963-42-7 MF C29 H28 F2 N6 O2 MW 530.57 2-Propenamide, N-[3-[2-[[2,3-difluoro-4-[4-(2-hydroxyethyl)-1-piperazinyl]phenyl]amino]-8-quinazolinyl]phenyl]- N-[3-[2-[[2,3-Difluoro-4-[4-(2-hydroxyethyl)piperazin-1-yl]phenyl]amino]quinazolin-8-yl]phenyl]acrylamide N-(3-(2-((2,3-Difluoro-4-(4-(2-hydroxyethyl)piperazin-1-yl)phenyl)amino)quinazolin-8-yl)phenyl)acrylamide EGFR-IN-3 UNII-708TLB8J3Y 708TLB8J3Y AK543910 Suzhou NeuPharma (Originator) Checkpoint Therapeutics Non-Small Cell Lung Cancer Therapy Solid Tumors Therapy PHASE 2 Checkpoint Therapeutics, Cancer, lung (non-small cell) (NSCLC), solid tumour RX518(CK-101) is an orally available third-generation and selective inhibitor of certain epidermal growth factor …

FDA approves third oncology drug Rozlytrek (entrectinib) that targets a key genetic driver of cancer, rather than a specific type of tumor

It’s only fair to share… FDA approves third oncology drug Rozlytrek (entrectinib) that targets a key genetic driver of cancer, rather than a specific type of tumor  FDA also approves drug for second indication in a type of lung cancer The U.S. Food and Drug Administration today granted accelerated approval to Rozlytrek (entrectinib), a treatment for …

SELPERCATINIB

It’s only fair to share… SELPERCATINIB LOXO 292 CAS: 2152628-33-4 Chemical Formula: C29H31N7O3 Molecular Weight: 525.613 CEGM9YBNGD UNII-CEGM9YBNGD  6-(2-hydroxy-2-methylpropoxy)-4-(6-{6-[(6-methoxypyridin- 3-yl)methyl]-3,6-diazabicyclo[3.1.1]heptan-3-yl}pyridin-3- yl)pyrazolo[1,5-a]pyridine-3-carbonitrile Selpercatinib is a tyrosine kinase inhibitor with antineoplastic properties. A phase I/II trial is also under way in pediatric patients and young adults with activating RET alterations and advanced solid or primary CNS tumors. …

FDA approves first treatment Ruzurgi (amifampridine) for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder

It’s only fair to share… FDA approves first treatment Ruzurgi (amifampridine)  for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder The U.S. Food and Drug Administration today approved Ruzurgi (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age. This is the first FDA …

Cladribine, クラドリビン

It’s only fair to share… Cladribine クラドリビン Leustatin クラドリビン RWJ 26251 / RWJ-26251 Molecular FormulaC10H12ClN5O3 Average mass285.687 Da 2-chloro-6-amino-9-(2-deoxy-β-D-erythro-pentofuranosyl)purine 2-Chlorodeoxyadenosine 4291-63-8 [RN] 6997 adenosine, 2-chloro-2′-deoxy- [ACD/Index Name] AU7357560 CDA (2R,3S,5R)-5-(6-Amino-2-chlor-9H-purin-9-yl)-2-(hydroxymethyl)tetrahydrofuran-3-ol Leustatin (Trade name) Litak (Trade name) MLS000759397 Movectro (Trade name) Mylinax QA-1968 LAUNCHED, 1993, USA Ortho Biotech, Janssen Biotech Cladribine, sold under the brand name Leustatin and Mavenclad among others, is a medication used to …

Macimorelin acetate

It’s only fair to share… Macimorelin Molecular FormulaC26H30N6O3 Average mass474.555 Da CAS  381231-18-1 Chemical Formula: C26H30N6O3 Exact Mass: 474.23794 Molecular Weight: 474.55480 Elemental Analysis: C, 65.80; H, 6.37; N, 17.71; O, 10.11 2-Methylalanyl-N-[(1R)-1-formamido-2-(1H-indol-3-yl)ethyl]-D-tryptophanamide 381231-18-1 [RN] 8680B21W73 9073 D-Tryptophanamide, 2-methylalanyl-N-[(1R)-1-(formylamino)-2-(1H-indol-3-yl)ethyl]- CAS 945212-59-9 (Macimorelin acetate) (2R)-2-(2-amino-2-methylpropanamido)-3-(1H-indol-3-yl)-N-[(1R)-2-(1H-indol-3-yl)-1-formamidoethyl]propanamide; acetic acid AEZS-130 ARD-07 D-87875 EP-01572 EP-1572 JMV-1843 USAN (ab-26) MACIMORELIN ACETATE AQZ1003RMG …

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 …