432:
409:
51:
694:
codon mutation for cystic fibrosis demonstrated increased CFTR protein production and function. Extending on this work, a mechanistic study with yeast and human cells has elucidated the details of ataluren-mediated nonstandard codon-anticodon base pairings which result in specific amino acid substitutions at specific codon positions in the CFTR protein.
42:
2183:
753:(EMA) and received market authorization from the European Commission to treat people with nonsense mutation Duchenne muscular dystrophy in August 2014; a confirmatory phase III clinical trial was required. By December it was on the market in Germany, France, Italy, Denmark, Spain and a number of other European Union countries.
693:
Studies have demonstrated that ataluren treatment increases expression of full-length dystrophin protein in human and mouse primary muscle cells containing the premature stop codon mutation for
Duchenne muscular dystrophy and rescues striated muscle function. Studies in mice with the premature stop
763:
In July 2016, NHS England agreed a
Managed Access Agreement (MAA) for Translarna providing reimbursed patient access to Translarna in England via a five-year MAA. This followed a positive recommendation from the National Institute for Health and Care Excellence (NICE) in April 2016, subject to PTC
1447:
Haas M, Vlcek V, Balabanov P, Salmonson T, Bakchine S, Markey G, et al. (January 2015). "European
Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene".
697:
The
European Medicines Agency review on the approval of ataluren concluded that "the non-clinical data available were considered sufficient to support the proposed mechanism of action and to alleviate earlier concerns on the selectivity of ataluren for premature stop codons."
618:
More than 10% of people taking ataluren in clinical trials experienced vomiting; more than 5% experienced diarrhea, nausea, headache, upper abdominal pain, and flatulence; between 1% and 5% of people experienced decreased appetite and weight loss,
1902:
741:
In 2010, PTC Therapeutics released preliminary results of its phase IIb clinical trial for
Duchenne muscular dystrophy, with participants not showing a significant improvement in the six minute walk distance after the 48 weeks of the trial.
1895:
1547:
730:
to identify compounds that increased the amount of protein expressed by mutated genes, and then optimized one of the hits in the screen to create this drug. As with the results of many cell-based screens, the
1581:
1888:
1548:"PTC Therapeutics, Inc. and Genzyme Corporation Announce Preliminary Results from the Phase 2b Clinical Trial of Ataluren; Primary Endpoint Does Not Reach Statistical Significance within Duration of Study"
689:
processing, stability of the mRNA or the resultant protein, thereby making a functional protein similar to the non-mutated endogenous product. It seems to work particularly well for the stop codon 'UGA'.
1022:
1245:
1055:
1614:
1862:
623:, high blood pressure, cough, nosebleeds, abdominal discomfort, constipation, rashes, pain in their arms, legs, and chest muscles, blood in their urine, urinary incontinence, and fever.
113:
562:
662:
of ataluren, it was discovered to bind and stabilize firefly luciferase, thus explaining the mechanism by which it created a false positive effect on the read through assay.
764:
and NHS England finalizing the terms of the MAA. NICE issued its final guidance later in July with implementation of the MAA for patients following within two months.
1573:
781:
746:
1014:
81:
1827:
1645:
1801:
518:
1521:
1337:
Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, et al. (May 2007). "PTC124 targets genetic disorders caused by nonsense mutations".
760:
for ataluren, which was based on a clinical trial in which ataluren missed its primary endpoint; PTC appealed and the FDA declined again in
October 2016.
1047:
1606:
1854:
788:
recommended not renewing the conditional marketing authorization for
Translarna, a medicine for treating people with Duchenne muscular dystrophy.
1844:
Text was copied from this source which is copyright
European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
634:
should not be given to someone taking ataluren, as they interfere with its mechanism of action. Caution should be used with drugs that induce
2137:
943:
1962:
1685:
854:
1194:
2108:
538:
504:
1390:"PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model"
722:'s lab at the University of Pennsylvania, which was initially funded in part by Parent Project Muscular Dystrophy. The team used
186:
143:
818:
1754:"Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis"
767:
In March 2017, PTC terminated development of ataluren for cystic fibrosis due to lack of efficacy in the phase III trials.
2069:
2173:
1988:
1492:"Press release: PTC Therapeutics Announces $ 15.4 Million NIH Research Grant for Duchenne Muscular Dystrophy | Evaluate"
308:
2154:
579:
388:
2223:
1637:
2003:
1880:
848:
785:
750:
1668:"NHS England successfully negotiates access to new drug treatment for children with duchenne muscular dystrophy"
2208:
1993:
1015:"Researchers question 'read-through' mechanism of muscular dystrophy drug ataluren : Spoonful of Medicine"
682:
257:
2142:
2093:
1912:
1491:
99:
427:
2203:
878:"Therapeutic suppression of premature termination codons: mechanisms and clinical considerations (review)"
377:
2218:
620:
248:
1499:
1048:"Press Release: Questions Raised About Process Used to Identify Experimental Drug for Genetic Disease"
2213:
1401:
1346:
1286:
1209:
1147:
1090:
757:
723:
1855:"Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 June 2024"
594:
Ataluren is used in the
European Union to treat people with Duchenne muscular dystrophy who have a
404:
203:
106:
546:
InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)
1734:
1473:
1370:
1195:"Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124"
1116:
1828:"EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna"
1802:"EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna"
50:
1275:"Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression"
843:
1783:
1726:
1465:
1429:
1362:
1314:
1237:
1175:
1108:
995:
939:
933:
907:
732:
678:
659:
595:
357:
158:
63:
297:
1773:
1765:
1716:
1457:
1419:
1409:
1354:
1304:
1294:
1227:
1217:
1165:
1155:
1136:"Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression"
1098:
985:
975:
897:
889:
727:
583:
444:
212:
317:
2187:
2113:
1273:
Roy B, Friesen WJ, Tomizawa Y, Leszyk JD, Zhuo J, Johnson B, et al. (November 2016).
631:
1522:"PTC Therapeutics, Inc. Initiates Phase 2 Study Of PTC124 In Duchenne Muscular Dystrophy"
673:(an effect referred to as "read-through") by promoting insertion of certain near-cognate
1405:
1350:
1290:
1213:
1151:
1094:
431:
408:
2148:
2046:
1934:
1778:
1753:
1424:
1389:
1309:
1274:
1232:
1170:
1135:
990:
963:
902:
877:
2197:
1738:
1667:
686:
420:
237:
1477:
1120:
91:
2118:
1980:
1916:
1769:
1374:
1193:
Auld DS, Lovell S, Thorne N, Lea WA, Maloney DJ, Shen M, et al. (March 2010).
932:
Pace A, Buscemi S, Piccionello AP, Pibiri I (2015). Scriven EF, Ramsden CA (eds.).
674:
126:
121:
814:
718:
Ataluren was discovered by scientists at PTC Therapeutics in a collaboration with
980:
2054:
2036:
2016:
1957:
1574:"PTC Therapeutics Receives Positive Opinion from CHMP for Translarna (ataluren)"
719:
1394:
Proceedings of the
National Academy of Sciences of the United States of America
1279:
Proceedings of the National Academy of Sciences of the United States of America
1202:
Proceedings of the National Academy of Sciences of the United States of America
1140:
Proceedings of the National Academy of Sciences of the United States of America
2077:
2059:
2031:
2026:
2021:
2011:
1952:
1461:
707:
670:
599:
480:
288:
2123:
2103:
1414:
1299:
1222:
1160:
85:
1787:
1730:
1469:
1433:
1366:
1318:
1241:
1179:
1112:
999:
911:
893:
17:
666:
268:
33:
1388:
Du M, Liu X, Welch EM, Hirawat S, Peltz SW, Bedwell DM (February 2008).
1358:
1103:
1078:
277:
1970:
1925:
1607:"PTC Therapeutics Announces Launch of Translarna (ataluren) in Germany"
647:
223:
1944:
1721:
1704:
635:
368:
610:
People who are pregnant or breast feeding should not take ataluren.
41:
938:. Advances in Heterocyclic Chemistry. Academic Press. p. 127.
337:
503:
494:
348:
643:
639:
328:
165:
152:
1884:
1496:
PTC, University of Pennsylvania, and the NIH via Evaluate Group
1686:"Drug Company Ends Ataluren Program for CF Nonsense Mutations"
393:
745:
In May 2014, ataluren received a positive opinion from the
756:
In February 2016, FDA declined to accept PTC Therapeutics
935:
3. Recent Advances in the Chemistry of 1,2,4-Oxadiazoles
1134:
Auld DS, Thorne N, Maguire WF, Inglese J (March 2009).
658:
While a large number of studies failed to identify the
2171:
817:. UK Electronic Medicines Compendium. 24 April 2017.
2086:
2068:
2045:
2002:
1979:
1943:
1924:
492:
479:
443:
438:
419:
387:
367:
347:
327:
307:
287:
267:
256:
247:
222:
202:
177:
142:
137:
112:
98:
80:
72:
62:
57:
602:gene, can walk, and are more than five years old.
838:
836:
815:"Translarna - Summary of Product Characteristics"
236:
211:
1752:Aslam AA, Sinha IP, Southern KW (March 2023).
782:Committee for Medicinal Products for Human Use
747:Committee for Medicinal Products for Human Use
1896:
1332:
1330:
1328:
8:
32:
1758:The Cochrane Database of Systematic Reviews
1268:
1266:
964:"A read-through drug put through its paces"
882:International Journal of Molecular Medicine
1903:
1889:
1881:
430:
407:
296:
49:
40:
1777:
1720:
1601:
1599:
1423:
1413:
1308:
1298:
1231:
1221:
1169:
1159:
1102:
1042:
1040:
989:
979:
957:
955:
901:
809:
807:
805:
803:
801:
738:Phase I clinical trials started in 2004.
316:
1638:"FDA snubs PTC appeal for Duchenne drug"
1052:NIH via Drug Discovery & Development
2178:
1079:"Chemical biology: ignore the nonsense"
797:
710:; its chemical name is 3-benzoic acid.
681:with no apparent effects on downstream
578:, is a medication for the treatment of
543:
523:
403:
276:
191:
927:
925:
923:
921:
421:
31:
27:Duchenne muscular dystrophy medication
1617:from the original on 27 December 2014
1019:Nature Medicine: Spoonful of Medicine
376:
356:
90:
7:
1648:from the original on 28 January 2017
857:from the original on 29 October 2020
125:
1502:from the original on 23 August 2023
1251:from the original on 23 August 2023
1058:from the original on 7 October 2017
876:Karijolich J, Yu YT (August 2014).
336:
227:
526:Fc3ccccc3c1nc(no1)c2cc(ccc2)C(=O)O
25:
1911:Other drugs for disorders of the
1865:from the original on 12 July 2024
1077:Schmitz A, Famulok M (May 2007).
821:from the original on 15 July 2017
2181:
1636:Pagliarulo N (17 October 2016).
1584:from the original on 30 May 2019
1528:(Press release). 27 January 2006
1025:from the original on 16 May 2017
464:
461:
455:
551:Key:OOUGLTULBSNHNF-UHFFFAOYSA-N
2070:Histone deacetylase inhibitors
1770:10.1002/14651858.CD012040.pub3
470:
449:
1:
1554:(Press release). 3 March 2010
1989:Delandistrogene moxeparvovec
981:10.1371/journal.pbio.1001458
669:less sensitive to premature
665:Ataluren is thought to make
638:, or that are substrates of
621:high levels of triglycerides
574:, sold under the brand name
962:Roberts RG (25 June 2013).
580:Duchenne muscular dystrophy
2240:
2004:Antisense oligonucleotides
1703:DeFrancesco L (May 2017).
1690:Cystic Fibrosis Foundation
735:of ataluren is not known.
439:Chemical and physical data
2132:
1859:European Medicines Agency
1832:European Medicines Agency
1806:European Medicines Agency
1462:10.1016/j.nmd.2014.11.011
1013:Devitt L (25 June 2013).
849:European Medicines Agency
786:European Medicines Agency
751:European Medicines Agency
559:
534:
514:
182:
48:
39:
1994:Onasemnogene abeparvovec
1965:clostridium histolyticum
92:International Drug Names
1913:musculo-skeletal system
1450:Neuromuscular Disorders
1415:10.1073/pnas.0711795105
1300:10.1073/pnas.1605336113
1223:10.1073/pnas.0909141107
1161:10.1073/pnas.0813345106
894:10.3892/ijmm.2014.1809
1705:"Drug pipeline: 1Q17"
853:. 17 September 2018.
582:. It was designed by
1709:Nature Biotechnology
758:new drug application
724:phenotypic screening
1808:. 15 September 2023
1613:. 3 December 2014.
1406:2008PNAS..105.2064D
1359:10.1038/nature05756
1351:2007Natur.447...87W
1291:2016PNAS..11312508R
1285:(44): 12508–12513.
1214:2010PNAS..107.4878A
1152:2009PNAS..106.3585A
1104:10.1038/nature05715
1095:2007Natur.447...42S
1054:. 3 February 2009.
771:Society and culture
161:(Prescription only)
36:
2159:Never to phase III
780:In June 2024, the
2224:Muscle protectors
2169:
2168:
1208:(11): 4878–4883.
945:978-0-12-802874-2
844:"Translarna EPAR"
733:biological target
660:biological target
606:Contraindications
596:nonsense mutation
569:
568:
505:Interactive image
389:CompTox Dashboard
169:
156:
16:(Redirected from
2231:
2186:
2185:
2184:
2177:
2094:Aceneuramic acid
1905:
1898:
1891:
1882:
1875:
1874:
1872:
1870:
1861:. 28 June 2024.
1851:
1845:
1843:
1841:
1839:
1824:
1818:
1817:
1815:
1813:
1798:
1792:
1791:
1781:
1749:
1743:
1742:
1724:
1722:10.1038/nbt.3874
1700:
1694:
1693:
1682:
1676:
1675:
1664:
1658:
1657:
1655:
1653:
1633:
1627:
1626:
1624:
1622:
1603:
1594:
1593:
1591:
1589:
1570:
1564:
1563:
1561:
1559:
1544:
1538:
1537:
1535:
1533:
1518:
1512:
1511:
1509:
1507:
1498:. 10 July 2007.
1488:
1482:
1481:
1444:
1438:
1437:
1427:
1417:
1400:(6): 2064–2069.
1385:
1379:
1378:
1334:
1323:
1322:
1312:
1302:
1270:
1261:
1260:
1258:
1256:
1250:
1235:
1225:
1199:
1190:
1184:
1183:
1173:
1163:
1146:(9): 3585–3590.
1131:
1125:
1124:
1106:
1074:
1068:
1067:
1065:
1063:
1044:
1035:
1034:
1032:
1030:
1010:
1004:
1003:
993:
983:
959:
950:
949:
929:
916:
915:
905:
873:
867:
866:
864:
862:
840:
831:
830:
828:
826:
811:
728:chemical library
584:PTC Therapeutics
565:
507:
487:
472:
466:
463:
457:
451:
434:
423:
412:
411:
397:
395:
380:
360:
340:
320:
300:
280:
260:
240:
230:
229:
215:
167:
164:
154:
151:
129:
94:
53:
44:
37:
35:
21:
2239:
2238:
2234:
2233:
2232:
2230:
2229:
2228:
2209:Cystic fibrosis
2194:
2193:
2192:
2182:
2180:
2172:
2170:
2165:
2164:
2149:Clinical trials
2128:
2114:Hyaluronic acid
2082:
2064:
2047:Glucocorticoids
2041:
1998:
1975:
1939:
1928:and derivatives
1920:
1909:
1879:
1878:
1868:
1866:
1853:
1852:
1848:
1837:
1835:
1826:
1825:
1821:
1811:
1809:
1800:
1799:
1795:
1764:(3): CD012040.
1751:
1750:
1746:
1702:
1701:
1697:
1692:. 3 March 2017.
1684:
1683:
1679:
1666:
1665:
1661:
1651:
1649:
1635:
1634:
1630:
1620:
1618:
1605:
1604:
1597:
1587:
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1572:
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1567:
1557:
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1546:
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1541:
1531:
1529:
1520:
1519:
1515:
1505:
1503:
1490:
1489:
1485:
1446:
1445:
1441:
1387:
1386:
1382:
1345:(7140): 87–91.
1336:
1335:
1326:
1272:
1271:
1264:
1254:
1252:
1248:
1197:
1192:
1191:
1187:
1133:
1132:
1128:
1089:(7140): 42–43.
1076:
1075:
1071:
1061:
1059:
1046:
1045:
1038:
1028:
1026:
1012:
1011:
1007:
974:(6): e1001458.
961:
960:
953:
946:
931:
930:
919:
875:
874:
870:
860:
858:
842:
841:
834:
824:
822:
813:
812:
799:
794:
778:
773:
716:
706:Ataluren is an
704:
679:nonsense codons
677:at the site of
656:
632:Aminoglycosides
629:
616:
614:Adverse effects
608:
592:
563:(what is this?)
560:
555:
552:
547:
542:
541:
530:
527:
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521:
510:
485:
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133:
101:
28:
23:
22:
15:
12:
11:
5:
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2106:
2101:
2096:
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2074:
2072:
2066:
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2062:
2057:
2051:
2049:
2043:
2042:
2040:
2039:
2034:
2029:
2024:
2019:
2014:
2008:
2006:
2000:
1999:
1997:
1996:
1991:
1985:
1983:
1981:Gene therapies
1977:
1976:
1974:
1973:
1968:
1960:
1955:
1949:
1947:
1941:
1940:
1938:
1937:
1931:
1929:
1922:
1921:
1910:
1908:
1907:
1900:
1893:
1885:
1877:
1876:
1846:
1834:. 28 June 2024
1819:
1793:
1744:
1695:
1677:
1659:
1642:BioPharma Dive
1628:
1595:
1565:
1539:
1513:
1483:
1439:
1380:
1324:
1262:
1185:
1126:
1069:
1036:
1005:
951:
944:
917:
888:(2): 355–362.
868:
832:
796:
795:
793:
790:
777:
774:
772:
769:
749:(CHMP) of the
715:
712:
703:
700:
655:
652:
628:
625:
615:
612:
607:
604:
591:
588:
567:
566:
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220:
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194:3-benzoic acid
193:
185:
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162:
148:
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140:
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102:administration
96:
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437:
433:
429:
426:
424:
422:ECHA InfoCard
418:
410:
406:
405:DTXSID5046776
402:
401:
399:
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386:
379:
375:
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234:
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192:
188:
181:
176:
170: Rx-only
163:
160:
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136:
128:
123:
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115:
111:
108:
105:
103:
97:
93:
89:
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75:
71:
67:
65:
61:
58:Clinical data
56:
52:
47:
43:
38:
30:
19:
2219:Orphan drugs
2119:Palovarotene
2109:Chondrocytes
2098:
1964:
1963:Collagenase
1935:Hydroquinine
1867:. Retrieved
1858:
1849:
1836:. Retrieved
1831:
1822:
1810:. Retrieved
1805:
1796:
1761:
1757:
1747:
1712:
1708:
1698:
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1671:
1662:
1650:. Retrieved
1641:
1631:
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1610:
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1568:
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1278:
1253:. Retrieved
1205:
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1129:
1086:
1082:
1072:
1060:. Retrieved
1051:
1027:. Retrieved
1018:
1008:
971:
968:PLOS Biology
967:
934:
885:
881:
871:
861:29 September
859:. Retrieved
847:
823:. Retrieved
779:
776:Legal status
766:
762:
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664:
657:
654:Pharmacology
630:
627:Interactions
617:
609:
593:
575:
571:
570:
561:
378:ChEMBL256997
144:Legal status
138:Legal status
29:
2214:Oxadiazoles
2145:from market
2055:Deflazacort
2037:Viltolarsen
2017:Drisapersen
1958:Chymopapain
1672:NHS England
1621:27 December
1611:MarketWatch
1578:MarketWatch
1456:(1): 5–13.
720:Lee Sweeney
671:stop codons
590:Medical use
488: g·mol
428:100.132.097
358:CHEBI:94805
213:775304-57-9
178:Identifiers
73:Other names
64:Trade names
2198:Categories
2078:Givinostat
2060:Vamorolone
2032:Nusinersen
2027:Golodirsen
2022:Eteplirsen
2012:Casimersen
1953:Bromelains
1715:(5): 400.
1255:4 November
792:References
708:oxadiazole
600:dystrophin
576:Translarna
493:3D model (
481:Molar mass
318:K16AME9I3V
289:ChemSpider
249:IUPHAR/BPS
204:CAS Number
187:IUPAC name
68:Translarna
18:Translarna
2155:Phase III
2143:Withdrawn
2124:Risdiplam
2104:Branaplam
1739:205284732
1558:22 August
1532:22 August
702:Chemistry
667:ribosomes
100:Routes of
86:Drugs.com
2188:Medicine
2099:Ataluren
1863:Archived
1788:36866921
1731:28486449
1646:Archived
1615:Archived
1582:Archived
1552:BioSpace
1526:BioSpace
1500:Archived
1478:41468577
1470:25497400
1434:18272502
1367:17450125
1319:27702906
1246:Archived
1242:20194791
1180:19208811
1121:29789135
1113:17450128
1056:Archived
1023:Archived
1000:23824301
912:24939317
855:Archived
819:Archived
572:Ataluren
269:DrugBank
238:11219835
114:ATC code
107:By mouth
34:Ataluren
1971:Trypsin
1945:Enzymes
1926:Quinine
1869:12 July
1838:29 June
1812:29 June
1779:9983356
1652:18 June
1506:18 June
1425:2538881
1402:Bibcode
1375:4423529
1347:Bibcode
1310:5098639
1287:Bibcode
1233:2841876
1210:Bibcode
1171:2638738
1148:Bibcode
1091:Bibcode
1062:18 June
1029:18 June
991:3692443
903:4094583
825:18 June
784:of the
714:History
648:OATP1B3
598:in the
486:284.246
445:Formula
298:9394889
278:DB05016
224:PubChem
130:)
124: (
122:M09AX03
2174:Portal
2138:WHO-EM
1786:
1776:
1737:
1729:
1588:30 May
1476:
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1432:
1422:
1373:
1365:
1339:Nature
1317:
1307:
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1230:
1178:
1168:
1119:
1111:
1083:Nature
998:
988:
942:
910:
900:
636:UGT1A9
519:SMILES
369:ChEMBL
338:D09323
157:
76:PTC124
2087:Other
1735:S2CID
1474:S2CID
1371:S2CID
1249:(PDF)
1198:(PDF)
1117:S2CID
851:(EMA)
726:of a
646:, or
539:InChI
495:JSmol
349:ChEBI
1871:2024
1840:2024
1814:2024
1784:PMID
1762:2023
1727:PMID
1654:2017
1623:2014
1590:2019
1560:2023
1534:2023
1508:2017
1466:PMID
1430:PMID
1363:PMID
1315:PMID
1257:2018
1238:PMID
1176:PMID
1109:PMID
1064:2017
1031:2017
996:PMID
940:ISBN
908:PMID
863:2020
827:2017
687:mRNA
675:tRNA
644:OAT3
640:OAT1
329:KEGG
309:UNII
258:7341
82:AHFS
1917:M09
1774:PMC
1766:doi
1717:doi
1458:doi
1420:PMC
1410:doi
1398:105
1355:doi
1343:447
1305:PMC
1295:doi
1283:113
1228:PMC
1218:doi
1206:107
1166:PMC
1156:doi
1144:106
1099:doi
1087:447
986:PMC
976:doi
898:PMC
890:doi
394:EPA
228:CID
159:POM
127:WHO
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