Category «orphan drug»

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 …

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, …

Voretigene neparvovec , ボレチジーンネパルボベック;

It’s only fair to share… Voretigene neparvovec Voretigene neparvovec-rzyl; Luxturna (TN) ボレチジーンネパルボベック; DNA (synthetic adeno-associated virus 2 vector AAV2-hRPE65v2) CAS: 1646819-03-5 2017/12/19, FDA  Luxturna, SPARK THERAPEUTICS Vision loss treatment, Retinal dystrophy AAV2-hRPE65v2 AAV2.RPE65 LTW-888 SPK-RPE65 rAAV.hRPE65v2 rAAV2-CBSB-hRPE65 2SPI046IKD (UNII code) melting point (°C) 72-90ºC Rayaprolu V. et al. J. Virol. vol. 87. no. 24. (2013)   FDA …