Category «orphan drug»

FDA approves first treatment Soliris (eculizumab) for neuromyelitis optica spectrum disorder, a rare autoimmune disease of the central nervous system

It’s only fair to share… The U.S. Food and Drug Administration today approved Soliris (eculizumab) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. NMOSD is an autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal …

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 …

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 …

Deutivacaftor

It’s only fair to share… Deutivacaftor RN: 1413431-07-8 UNII: SHA6U5FJZL,  N-[2-tert-butyl-4-[1,1,1,3,3,3-hexadeuterio-2-(trideuteriomethyl)propan-2-yl]-5-hydroxyphenyl]-4-oxo-1H-quinoline-3-carboxamide Molecular Formula, C24-H28-N2-O3, Molecular Weight, 401.552 Synonyms CTP-656 D9-ivacaftor Deutivacaftor Ivacaftor D9 UNII-SHA6U5FJZL VX-561 WHO 10704 Treatment of Cystic Fibrosis Originator Concert Pharmaceuticals Class Amides; Aminophenols; Antifibrotics; Organic deuterium compounds; Quinolones; Small molecules Mechanism of Action Cystic fibrosis transmembrane conductance regulator stimulants Orphan Drug Status Yes – Cystic fibrosis Phase II Cystic …

Solriamfetol hydrochloride, ソルリアムフェトル塩酸塩 , солриамфетол , سولريامفيتول , 索安非托 ,

It’s only fair to share… Solriamfetol hydrochloride FDA APPROVED 2019/3/20, Sunosi ソルリアムフェトル塩酸塩; R228060, R 228060 Formula C10H14N2O2. HCl CAS 178429-65-7 HCL Mol weight 230.6913 (2R)-2-Amino-3-phenylpropyl carbamate (2R)-2-Amino-3-phenylpropylcarbamat 10117 178429-62-4 [RN] FREE FORM Benzenepropanol, β-amino-, carbamate (ester), (βR)- [ солриамфетол [Russian] [INN] سولريامفيتول [Arabic] [INN] 索安非托 [Chinese] [INN] JZP-110 Originator SK Holdings Developer Jazz Pharmaceuticals plc; SK biopharmaceuticals Class Carbamates; Sleep disorder therapies; Small molecules Mechanism of Action Adrenergic uptake inhibitors; Dopamine uptake …

RISDIPLAM , リスジプラム

It’s only fair to share… RISDIPLAM RG-7916, RO-7034067, リスジプラム Formula C22H23N7O Cas 1825352-65-5 Mol weight 401.4643 US9969754 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one   WHO 10614 RG-7916 HY-109101 RO7034067 CS-0039501 EX-A2074 RG7916 The compound was originally claimed in WO2015173181 , for treating spinal muscular atrophy (SMA). Roche , under license from PTC Therapeutics , and Chugai , are developing risdiplam (RO-7034067; RG-7916), …

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 …