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weeks. Of the research participants, 53% were male, 97% were white, and the mean age was 24 years at study entry. Nine (18%) patients were younger than age 18. The primary objective was to evaluate the change in the modified
Friedreich's Ataxia Rating Scale (mFARS) score compared to placebo at week 48. The mFARS is a clinical assessment that measures disease progression, namely swallowing and speech (bulbar), upper limb coordination, lower limb coordination, and upright stability. Individuals receiving omaveloxolone performed better on the mFARS than people receiving placebo.
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Friedreich's ataxia causes progressive damage to the spinal cord, peripheral nerves, and the brain, resulting in uncoordinated muscle movement, poor balance, difficulty walking, changes in speech and swallowing, and a shortened lifespan. The condition can also cause heart disease. This disease tends
753:
adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product
Skyclarys, intended for the treatment of Friedreich's ataxia. The applicant for this medicinal product is Reata Ireland Limited. Omaveloxolone was approved for medical use in the European
709:
The efficacy and safety of omaveloxolone was evaluated in a 48-week randomized, placebo-controlled, and double-blind study and an open-label extension. Study 1 enrolled 103 individuals with
Friedreich's ataxia who received placebo (52 individuals) or omaveloxolone 150 mg (51 individuals) for 48
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The most common side effects include an increase in alanine transaminase and an increase of aspartate aminotransferase, which can be signs of liver damage, headache, nausea, abdominal pain, fatigue, diarrhea and musculoskeletal pain.
646:), which provide a specific and finite antioxidative potential, omaveloxolone, through Nrf2, broadly activates intracellular and mitochondrial antioxidative pathways, in addition to pathways that may directly increase
510:
InChI=1S/C32H43NO4/c1-27(2)11-13-32(26(36)37-8)14-12-31(7)24(20(32)17-27)21(34)15-23-29(5)16-19(18-33)25(35)28(3,4)22(29)9-10-30(23,31)6/h15-16,20,22,24H,9-14,17H2,1-8H3/t20-,22-,24-,29-,30+,31+,32-/m0/s1
194:
N-((4aS,6aR,6bS,8aR,12aS,14aR,14bS)-1 1-cyano-2,2,6a,6b,9,9,12a-heptamethyl-10,14-dioxo-1,2,3,4,4a,5,6,6a,6b,7,8,8a,9,10,12a,14,14a,14b- octadecahydropicen-4a-yl)-2,2-difluoropropanamide
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Reisman SA, Lee CY, Meyer CJ, Proksch JW, Ward KW (July 2014). "Topical application of the synthetic triterpenoid RTA 408 activates Nrf2 and induces cytoprotective genes in rat skin".
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The mechanism of action of omaveloxolone and its related compounds has been demonstrated to be through a combination of activation of the antioxidative transcription factor
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transcriptionally regulates multiple genes that play both direct and indirect roles in producing antioxidative potential and the production of cellular energy (
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959:"Reata Pharmaceuticals Announces FDA Approval of Skyclarys (Omavaloxolone), the First and Only Drug Indicated for Patients with Friedreich's Ataxia"
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Although rare, Friedreich's ataxia is the most common form of hereditary ataxia in the United States, affecting about one in every 50,000 people.
1181:"Neuroprotective effect of Nrf2/ARE activators, CDDO ethylamide and CDDO trifluoroethylamide, in a mouse model of amyotrophic lateral sclerosis"
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Text was copied from this source which is copyright
European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
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Omaveloxolone was approved for medical use in the United States in
February 2023, and in the European Union in February 2024. The US
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Subramony SH, Lynch DL (May 2023). "A Milestone in the
Treatment of Ataxias: Approval of Omaveloxolone for Friedreich Ataxia".
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Omaveloxolone is a second generation member of the synthetic oleanane triterpenoid compounds and in clinical development by
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1230:"Topical application of the synthetic triterpenoid RTA 408 protects mice from radiation-induced dermatitis"
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Neymotin A, Calingasan NY, Wille E, Naseri N, Petri S, Damiano M, et al. (July 2011).
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686:. Omaveloxolone is under clinical investigation for a variety of indications, including
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Omaveloxolone was approved for medical use in the United States in
February 2023.
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O=C4C(\C#N)=C/5(/C3=C/C(=O)2(CC1(NC(=O)C(F)(F)C)CCC(C)(C)C12)(C)3(C)CC5C4(C)C)C
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Reisman SA, Lee CY, Meyer CJ, Proksch JW, Sonis ST, Ward KW (May 2014).
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for "RTA 408 Capsules in
Patients With Friedreich's Ataxia - MOXIe" at
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to develop in children and teenagers and gradually worsens over time.
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This article incorporates text from this source, which is in the
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and inhibition of the pro-inflammatory transcription factor
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717:(FDA) granted the application for omaveloxolone
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674:have demonstrated that omaveloxolone possesses
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747:Committee for Medicinal Products for Human Use
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729:, and rare pediatric disease designations.
589:for the treatment of Friedreich's ataxia.
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682:activities and the ability to improve
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832:from the original on 15 December 2023
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1111:from the original on 10 January 2024
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860:Union Register of medicinal products
1197:10.1016/j.freeradbiomed.2011.03.027
1185:Free Radical Biology & Medicine
1135:Archives of Dermatological Research
965:(Press release). 28 February 2023.
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783:"Skyclarys- omaveloxolone capsule"
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969:from the original on 1 March 2023
929:from the original on 1 March 2023
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793:from the original on 1 July 2023
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1094:New Drug Therapy Approvals 2023
990:"Omaveloxolone: First Approval"
856:"Skyclarys product information"
515:Key:WPTTVJLTNAWYAO-KPOXMGGZSA-N
1333:Drugs not assigned an ATC code
434:
410:
1:
1103:Food and Drug Administration
921:Food and Drug Administration
715:Food and Drug Administration
622:, adenosine triphosphate or
571:Food and Drug Administration
546:, sold under the brand name
684:mitochondrial bioenergetics
573:(FDA) considers it to be a
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1061:10.1007/s12311-023-01568-8
1006:10.1007/s40265-023-01874-9
963:Reata Pharmaceuticals Inc.
554:used for the treatment of
400:Chemical and physical data
1147:10.1007/s00403-013-1433-7
828:(EMA). 14 December 2023.
826:European Medicines Agency
751:European Medicines Agency
575:first-in-class medication
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1107:(Report). January 2024.
754:Union in February 2024.
700:corneal endothelial cell
692:mitochondrial myopathies
648:mitochondrial biogenesis
1288:Clinical trial number
745:In December 2023, the
668:Reata Pharmaceuticals
925:. 28 February 2023.
698:, and prevention of
1246:2014RadR..181..512R
988:Lee A (June 2023).
733:Society and culture
688:Friedreich's ataxia
672:Preclinical studies
600:Mechanism of action
556:Friedreich's ataxia
25:
1296:ClinicalTrials.gov
1234:Radiation Research
862:. 12 February 2024
1255:10.1667/RR13578.1
680:anti-inflammatory
585:Omaveloxolone is
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1343:Orphan drugs
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738:Legal status
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628:mitochondria
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213:1474034-05-3
145:Legal status
139:Legal status
80:License data
18:
1353:Triterpenes
1291:NCT02255435
1032:16 December
973:28 February
933:28 February
866:19 February
836:16 December
797:16 December
719:orphan drug
452: g·mol
178:Identifiers
53:Other names
44:Trade names
1327:Categories
1049:Cerebellum
758:References
723:fast track
552:medication
457:3D model (
445:Molar mass
318:G69Z98951Q
289:ChemSpider
249:IUPHAR/BPS
204:CAS Number
187:IUPAC name
16:Medication
1115:9 January
1077:258843532
1022:258567442
650:(such as
640:vitamin E
587:indicated
548:Skyclarys
110:Routes of
72:Monograph
66:Drugs.com
48:Skyclarys
1348:Steroids
1317:Medicine
1272:23906747
1264:24720753
1215:21457778
1163:25733020
1155:24362512
1109:Archived
1069:37219716
1026:Archived
1014:37155124
967:Archived
927:Archived
830:Archived
791:Archived
787:DailyMed
560:by mouth
534:(verify)
298:34980948
269:DrugBank
238:71811910
124:ATC code
117:By mouth
94:DailyMed
1242:Bibcode
1206:3109235
713:The US
662:History
550:, is a
450:554.723
406:Formula
278:DB12513
224:PubChem
96::
56:RTA 408
1303:Portal
1270:
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1203:
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1153:
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1012:
654:) and
483:SMILES
338:D10964
160:℞-only
158:
92:
1268:S2CID
1159:S2CID
1105:(FDA)
1101:U.S.
1097:(PDF)
1073:S2CID
1018:S2CID
994:Drugs
923:(FDA)
919:U.S.
652:PGC1α
610:NF-κB
503:InChI
459:JSmol
349:ChEBI
1260:PMID
1211:PMID
1151:PMID
1117:2024
1065:PMID
1034:2023
1010:PMID
975:2023
935:2023
868:2024
838:2023
799:2023
678:and
636:e.g.
620:i.e.
616:Nrf2
606:Nrf2
329:KEGG
309:UNII
258:7573
131:None
62:AHFS
1250:doi
1238:181
1201:PMC
1193:doi
1143:doi
1139:306
1057:doi
1002:doi
642:or
624:ATP
374:EPA
228:CID
1329::
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634:(
461:)
438:3
435:O
432:2
429:N
426:2
423:F
417:H
411:C
376:)
372:(
168::
156::
64:/
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