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class="vector-toc-list"> </ul> </li> </ul> </div> </div> </nav> </div> </div> <div class="mw-content-container"> <main id="content" class="mw-body"> <header class="mw-body-header vector-page-titlebar"> <nav aria-label="Contents" class="vector-toc-landmark"> <div id="vector-page-titlebar-toc" class="vector-dropdown vector-page-titlebar-toc vector-button-flush-left" > <input type="checkbox" id="vector-page-titlebar-toc-checkbox" role="button" aria-haspopup="true" data-event-name="ui.dropdown-vector-page-titlebar-toc" class="vector-dropdown-checkbox " aria-label="Toggle the table of contents" > <label id="vector-page-titlebar-toc-label" for="vector-page-titlebar-toc-checkbox" class="vector-dropdown-label cdx-button cdx-button--fake-button cdx-button--fake-button--enabled cdx-button--weight-quiet cdx-button--icon-only " aria-hidden="true" ><span class="vector-icon mw-ui-icon-listBullet mw-ui-icon-wikimedia-listBullet"></span> <span class="vector-dropdown-label-text">Toggle the table of contents</span> </label> <div class="vector-dropdown-content"> <div id="vector-page-titlebar-toc-unpinned-container" class="vector-unpinned-container"> </div> </div> </div> </nav> <h1 id="firstHeading" class="firstHeading mw-first-heading"><span class="mw-page-title-main">Migalastat</span></h1> <div id="p-lang-btn" class="vector-dropdown mw-portlet mw-portlet-lang" > <input type="checkbox" id="p-lang-btn-checkbox" role="button" aria-haspopup="true" data-event-name="ui.dropdown-p-lang-btn" class="vector-dropdown-checkbox mw-interlanguage-selector" aria-label="Go to an article in another language. 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div.hatnote{padding-left:1.6em;margin-bottom:0.5em}.mw-parser-output .hatnote i{font-style:normal}.mw-parser-output .hatnote+link+.hatnote{margin-top:-0.5em}@media print{body.ns-0 .mw-parser-output .hatnote{display:none!important}}</style><div role="note" class="hatnote navigation-not-searchable">Not to be confused with <a href="/wiki/Miglustat" title="Miglustat">miglustat</a>.</div> <style data-mw-deduplicate="TemplateStyles:r1257001546">.mw-parser-output .infobox-subbox{padding:0;border:none;margin:-3px;width:auto;min-width:100%;font-size:100%;clear:none;float:none;background-color:transparent}.mw-parser-output .infobox-3cols-child{margin:auto}.mw-parser-output .infobox .navbar{font-size:100%}@media screen{html.skin-theme-clientpref-night .mw-parser-output .infobox-full-data:not(.notheme)>div:not(.notheme)[style]{background:#1f1f23!important;color:#f8f9fa}}@media screen and (prefers-color-scheme:dark){html.skin-theme-clientpref-os .mw-parser-output .infobox-full-data:not(.notheme) div:not(.notheme){background:#1f1f23!important;color:#f8f9fa}}@media(min-width:640px){body.skin--responsive .mw-parser-output .infobox-table{display:table!important}body.skin--responsive .mw-parser-output .infobox-table>caption{display:table-caption!important}body.skin--responsive .mw-parser-output .infobox-table>tbody{display:table-row-group}body.skin--responsive .mw-parser-output .infobox-table tr{display:table-row!important}body.skin--responsive .mw-parser-output .infobox-table th,body.skin--responsive .mw-parser-output .infobox-table td{padding-left:inherit;padding-right:inherit}}</style><table class="infobox" style="border-spacing:2px;"><caption class="infobox-title"><span title="International nonproprietary name (INN): Migalastat">Migalastat</span></caption><tbody><tr><td colspan="2" class="infobox-image notheme" style="background-color: #f8f9fa;"><span typeof="mw:File"><a href="/wiki/File:1-Deoxygalactonojirimycin.svg" class="mw-file-description"><img src="//upload.wikimedia.org/wikipedia/commons/thumb/d/d2/1-Deoxygalactonojirimycin.svg/125px-1-Deoxygalactonojirimycin.svg.png" decoding="async" width="125" height="102" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/d/d2/1-Deoxygalactonojirimycin.svg/188px-1-Deoxygalactonojirimycin.svg.png 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/d/d2/1-Deoxygalactonojirimycin.svg/250px-1-Deoxygalactonojirimycin.svg.png 2x" data-file-width="512" data-file-height="416" /></a></span></td></tr><tr><th colspan="2" class="infobox-header" style="background:#ddd">Clinical data</th></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;">Pronunciation</th><td class="infobox-data">mi GAL a stat </td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Drug_nomenclature#Trade_names" title="Drug nomenclature">Trade names</a></th><td class="infobox-data">Galafold</td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;">Other names</th><td class="infobox-data">DDIG, AT1001, 1-deoxygalactonojirimycin</td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/American_Society_of_Health-System_Pharmacists" title="American Society of Health-System Pharmacists">AHFS</a>/<a href="/wiki/Drugs.com" title="Drugs.com">Drugs.com</a></th><td class="infobox-data"><span title="www.drugs.com"><a rel="nofollow" class="external text" href="https://www.drugs.com/monograph/migalastat-hydrochloride.html">Monograph</a></span></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Pregnancy_category" title="Pregnancy category">Pregnancy<br />category</a></th><td class="infobox-data"><style data-mw-deduplicate="TemplateStyles:r1126788409">.mw-parser-output .plainlist ol,.mw-parser-output .plainlist ul{line-height:inherit;list-style:none;margin:0;padding:0}.mw-parser-output .plainlist ol li,.mw-parser-output .plainlist ul li{margin-bottom:0}</style><div class="plainlist"> <ul><li><small><abbr class="country-name" title="Australia">AU</abbr>:</small> B3</li> <li class="mw-empty-elt"></li></ul> </div></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Route_of_administration" title="Route of administration">Routes of<br />administration</a></th><td class="infobox-data"><a href="/wiki/Oral_administration" title="Oral administration">By mouth</a></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Anatomical_Therapeutic_Chemical_Classification_System" title="Anatomical Therapeutic Chemical Classification System">ATC code</a></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><a href="/wiki/ATC_code_A16" title="ATC code A16">A16AX14</a> (<span title="www.whocc.no"><a rel="nofollow" class="external text" href="https://www.whocc.no/atc_ddd_index/?code=A16AX14">WHO</a></span>) </li></ul></div></td></tr><tr><th colspan="2" class="infobox-header" style="background:#ddd">Legal status</th></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Regulation_of_therapeutic_goods" title="Regulation of therapeutic goods">Legal status</a></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"> <ul><li><small><abbr class="country-name" title="Australia">AU</abbr>:</small> <a href="/wiki/Standard_for_the_Uniform_Scheduling_of_Medicines_and_Poisons#Schedule_4" title="Standard for the Uniform Scheduling of Medicines and Poisons">S4</a> (Prescription only)<sup id="cite_ref-1" class="reference"><a href="#cite_note-1"><span class="cite-bracket">[</span>1<span class="cite-bracket">]</span></a></sup><sup id="cite_ref-2" class="reference"><a href="#cite_note-2"><span class="cite-bracket">[</span>2<span class="cite-bracket">]</span></a></sup><sup id="cite_ref-3" class="reference"><a href="#cite_note-3"><span class="cite-bracket">[</span>3<span class="cite-bracket">]</span></a></sup></li> <li><small><abbr class="country-name" title="Canada">CA</abbr></small>: <a href="/wiki/Prescription_drug" title="Prescription drug"> ℞-only</a><sup id="cite_ref-4" class="reference"><a href="#cite_note-4"><span class="cite-bracket">[</span>4<span class="cite-bracket">]</span></a></sup></li> <li><small><abbr class="country-name" title="United States">US</abbr>:</small> <a href="/wiki/Prescription_drug" title="Prescription drug">℞-only</a></li> <li><small><abbr class="country-name" title="European Union">EU</abbr>:</small> Rx-only</li> <li>In general: ℞ (Prescription only)</li></ul></div> </td></tr><tr><th colspan="2" class="infobox-header" style="background:#ddd"><a href="/wiki/Pharmacokinetics" title="Pharmacokinetics">Pharmacokinetic</a> data</th></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Bioavailability" title="Bioavailability">Bioavailability</a></th><td class="infobox-data">75%</td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Plasma_protein_binding" title="Plasma protein binding">Protein binding</a></th><td class="infobox-data">None</td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Metabolite" title="Metabolite">Metabolites</a></th><td class="infobox-data"><i>O</i>-<a href="/wiki/Glucuronide" title="Glucuronide">glucuronides</a> (<15%)</td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Biological_half-life" title="Biological half-life">Elimination <span class="nowrap">half-life</span></a></th><td class="infobox-data">3–5 hours (single dose)</td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Excretion" title="Excretion">Excretion</a></th><td class="infobox-data"><a href="/wiki/Urine" title="Urine">Urine</a> (77%), <a href="/wiki/Feces" title="Feces">feces</a> (20%)</td></tr><tr><th colspan="2" class="infobox-header" style="background:#ddd">Identifiers</th></tr><tr><td colspan="2" class="infobox-full-data"><div class="collapsible-list mw-collapsible mw-collapsed" style="text-align: left;"> <div style="line-height: 1.6em; font-weight: bold;"><div><a href="/wiki/IUPAC_nomenclature_of_chemistry" title="IUPAC nomenclature of chemistry">IUPAC name</a></div></div> <ul class="mw-collapsible-content" style="margin-top: 0; margin-bottom: 0; line-height: inherit; list-style: none; margin-left: 0; word-break:break-all; text-align:left; padding-left:1.5em; text-indent:-1.5em;"><li style="line-height: inherit; margin: 0"><div style="font-size: 97%;">(2<i>R</i>,3<i>S</i>,4<i>R</i>,5<i>S</i>)-2-(Hydroxymethyl)-3,4,5-piperidinetriol</div></li></ul> </div></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/CAS_Registry_Number" title="CAS Registry Number">CAS Number</a></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><span title="commonchemistry.cas.org"><a rel="nofollow" class="external text" href="https://commonchemistry.cas.org/detail?cas_rn=108147-54-2">108147-54-2</a></span><sup> <span typeof="mw:File"><span><img alt="check" src="//upload.wikimedia.org/wikipedia/en/thumb/f/fb/Yes_check.svg/7px-Yes_check.svg.png" decoding="async" width="7" height="7" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/en/thumb/f/fb/Yes_check.svg/11px-Yes_check.svg.png 1.5x, //upload.wikimedia.org/wikipedia/en/thumb/f/fb/Yes_check.svg/14px-Yes_check.svg.png 2x" data-file-width="600" data-file-height="600" /></span></span><span style="display:none">Y</span></sup></li><li>HCl: <span title="commonchemistry.cas.org"><a rel="nofollow" class="external text" href="https://commonchemistry.cas.org/detail?cas_rn=75172-81-5">75172-81-5</a></span><sup> <span typeof="mw:File"><span><img alt="check" src="//upload.wikimedia.org/wikipedia/en/thumb/f/fb/Yes_check.svg/7px-Yes_check.svg.png" decoding="async" width="7" height="7" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/en/thumb/f/fb/Yes_check.svg/11px-Yes_check.svg.png 1.5x, //upload.wikimedia.org/wikipedia/en/thumb/f/fb/Yes_check.svg/14px-Yes_check.svg.png 2x" data-file-width="600" data-file-height="600" /></span></span><span style="display:none">Y</span></sup></li></ul></div></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/PubChem#CID" title="PubChem">PubChem</a> <span style="font-weight:normal"><abbr title="Compound ID">CID</abbr></span></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><span title="pubchem.ncbi.nlm.nih.gov"><a rel="nofollow" class="external text" href="https://pubchem.ncbi.nlm.nih.gov/compound/176077">176077</a></span></li></ul></div></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/DrugBank" title="DrugBank">DrugBank</a></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><span title="www.drugbank.ca"><a rel="nofollow" class="external text" href="https://www.drugbank.ca/drugs/DB05018">DB05018</a></span></li></ul></div></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/ChemSpider" title="ChemSpider">ChemSpider</a></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><span title="www.chemspider.com"><a rel="nofollow" class="external text" href="https://www.chemspider.com/Chemical-Structure.153388.html">153388</a></span></li></ul></div></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Unique_Ingredient_Identifier" title="Unique Ingredient Identifier">UNII</a></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><span title="precision.fda.gov"><a rel="nofollow" class="external text" href="https://precision.fda.gov/uniisearch/srs/unii/C4XNY919FW">C4XNY919FW</a></span></li><li>HCl: <span title="precision.fda.gov"><a rel="nofollow" class="external text" href="https://precision.fda.gov/uniisearch/srs/unii/CLY7M0XD20">CLY7M0XD20</a></span><sup> <span typeof="mw:File"><span><img alt="check" src="//upload.wikimedia.org/wikipedia/en/thumb/f/fb/Yes_check.svg/7px-Yes_check.svg.png" decoding="async" width="7" height="7" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/en/thumb/f/fb/Yes_check.svg/11px-Yes_check.svg.png 1.5x, //upload.wikimedia.org/wikipedia/en/thumb/f/fb/Yes_check.svg/14px-Yes_check.svg.png 2x" data-file-width="600" data-file-height="600" /></span></span><span style="display:none">Y</span></sup></li></ul></div></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/KEGG" title="KEGG">KEGG</a></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><span title="www.kegg.jp"><a rel="nofollow" class="external text" href="https://www.kegg.jp/entry/D10359">D10359</a></span></li><li>HCl: <span title="www.kegg.jp"><a rel="nofollow" class="external text" href="https://www.kegg.jp/entry/D05031">D05031</a></span></li></ul></div></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/ChEMBL" title="ChEMBL">ChEMBL</a></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><span title="www.ebi.ac.uk"><a rel="nofollow" class="external text" href="https://www.ebi.ac.uk/chembldb/index.php/compound/inspect/ChEMBL110458">ChEMBL110458</a></span></li></ul></div></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/CompTox_Chemicals_Dashboard" title="CompTox Chemicals Dashboard">CompTox Dashboard</a> <span style="font-weight:normal">(<abbr title="U.S. Environmental Protection Agency">EPA</abbr>)</span></th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><span title="comptox.epa.gov"><a rel="nofollow" class="external text" href="https://comptox.epa.gov/dashboard/chemical/details/DTXSID201022520">DTXSID201022520</a> <span class="mw-valign-text-top noprint" typeof="mw:File/Frameless"><a href="https://www.wikidata.org/wiki/Q161613#P3117" title="Edit this at Wikidata"><img alt="Edit this at Wikidata" src="//upload.wikimedia.org/wikipedia/en/thumb/8/8a/OOjs_UI_icon_edit-ltr-progressive.svg/10px-OOjs_UI_icon_edit-ltr-progressive.svg.png" decoding="async" width="10" height="10" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/en/thumb/8/8a/OOjs_UI_icon_edit-ltr-progressive.svg/15px-OOjs_UI_icon_edit-ltr-progressive.svg.png 1.5x, //upload.wikimedia.org/wikipedia/en/thumb/8/8a/OOjs_UI_icon_edit-ltr-progressive.svg/20px-OOjs_UI_icon_edit-ltr-progressive.svg.png 2x" data-file-width="20" data-file-height="20" /></a></span></span></li></ul></div></td></tr><tr><th colspan="2" class="infobox-header" style="background:#ddd">Chemical and physical data</th></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Chemical_formula" title="Chemical formula">Formula</a></th><td class="infobox-data"><span title="Carbon">C</span><sub>6</sub><span title="Hydrogen">H</span><sub>13</sub><span title="Nitrogen">N</span><span title="Oxygen">O</span><sub>4</sub></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;"><a href="/wiki/Molar_mass" title="Molar mass">Molar mass</a></th><td class="infobox-data"><span class="nowrap"><span data-sort-value="7002163173000000000♠"></span>163.173</span> g·mol<sup>−1</sup></td></tr><tr><th scope="row" class="infobox-label" style="line-height:1.2em;">3D model (<a href="/wiki/JSmol" class="mw-redirect" title="JSmol">JSmol</a>)</th><td class="infobox-data"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1126788409"><div class="plainlist"><ul><li><span title="chemapps.stolaf.edu (3D interactive model)"><a rel="nofollow" class="external text" href="https://chemapps.stolaf.edu/jmol/jmol.php?model=C1%5BC%40%40H%5D%28%5BC%40H%5D%28%5BC%40H%5D%28%5BC%40H%5D%28N1%29CO%29O%29O%29O">Interactive image</a></span></li></ul></div></td></tr><tr><td colspan="2" class="infobox-full-data"><div class="collapsible-list mw-collapsible mw-collapsed" style="text-align: left;"> <div style="line-height: 1.6em; font-weight: bold;"><div><a href="/wiki/Simplified_molecular-input_line-entry_system" class="mw-redirect" title="Simplified molecular-input line-entry system">SMILES</a></div></div> <ul class="mw-collapsible-content" style="margin-top: 0; margin-bottom: 0; line-height: inherit; list-style: none; margin-left: 0; word-break:break-all;"><li style="line-height: inherit; margin: 0"><div style="word-wrap:break-word; text-indent:-1.5em; text-align:left; padding-left:1.5em; font-size:97%; line-height:120%;">C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O</div></li></ul> </div></td></tr><tr><td colspan="2" class="infobox-full-data"><div class="collapsible-list mw-collapsible mw-collapsed" style="text-align: left;"> <div style="line-height: 1.6em; font-weight: bold;"><div><a href="/wiki/International_Chemical_Identifier" title="International Chemical Identifier">InChI</a></div></div> <ul class="mw-collapsible-content" style="margin-top: 0; margin-bottom: 0; line-height: inherit; list-style: none; margin-left: 0; word-break:break-all;"><li style="line-height: inherit; margin: 0"><div style="word-wrap:break-word; text-indent:-1.5em; text-align:left; padding-left:1.5em; font-size:97%; line-height:120%;">InChI=1S/C6H13NO4/c8-2-3-5(10)6(11)4(9)1-7-3/h3-11H,1-2H2/t3-,4+,5+,6-/m1/s1</div></li><li style="line-height: inherit; margin: 0"><div style="word-wrap:break-word; text-indent:-1.5em; text-align:left; padding-left:1.5em; font-size:97%; line-height:120%;">Key:LXBIFEVIBLOUGU-DPYQTVNSSA-N</div></li></ul> </div></td></tr></tbody></table> <p><b>Migalastat</b>, sold under the brand name <b>Galafold</b>, is a medication for the treatment of <a href="/wiki/Fabry_disease" title="Fabry disease">Fabry disease</a>, a rare genetic disorder. It was developed by <a href="/wiki/Amicus_Therapeutics" title="Amicus Therapeutics">Amicus Therapeutics</a>. The US <a href="/wiki/Food_and_Drug_Administration" title="Food and Drug Administration">Food and Drug Administration</a> (FDA) granted it <a href="/wiki/Orphan_drug" title="Orphan drug">orphan drug</a> status in 2004,<sup id="cite_ref-Orphans_5-0" class="reference"><a href="#cite_note-Orphans-5"><span class="cite-bracket">[</span>5<span class="cite-bracket">]</span></a></sup> and the European Commission followed in 2006.<sup id="cite_ref-6" class="reference"><a href="#cite_note-6"><span class="cite-bracket">[</span>6<span class="cite-bracket">]</span></a></sup> The European Medicines Agency's <a href="/wiki/Committee_for_Medicinal_Products_for_Human_Use" title="Committee for Medicinal Products for Human Use">Committee for Medicinal Products for Human Use</a> (CHMP) granted the drug a marketing approval under the name Galafold in May 2016.<sup id="cite_ref-Newswire_7-0" class="reference"><a href="#cite_note-Newswire-7"><span class="cite-bracket">[</span>7<span class="cite-bracket">]</span></a></sup><sup id="cite_ref-EPAR_8-0" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup><sup id="cite_ref-Galafold_EPAR_9-0" class="reference"><a href="#cite_note-Galafold_EPAR-9"><span class="cite-bracket">[</span>9<span class="cite-bracket">]</span></a></sup> </p><p>The U.S. <a href="/wiki/Food_and_Drug_Administration" title="Food and Drug Administration">Food and Drug Administration</a> (FDA) considers it to be a <a href="/wiki/First-in-class_medication" title="First-in-class medication">first-in-class medication</a>.<sup id="cite_ref-10" class="reference"><a href="#cite_note-10"><span class="cite-bracket">[</span>10<span class="cite-bracket">]</span></a></sup> </p> <meta property="mw:PageProp/toc" /> <div class="mw-heading mw-heading2"><h2 id="Medical_uses">Medical uses</h2><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=1" title="Edit section: Medical uses"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <p>Migalastat is used for the long-term treatment of Fabry disease in adults and adolescents aged 16 or older with an amenable mutation of the enzyme <a href="/wiki/Alpha-galactosidase" class="mw-redirect" title="Alpha-galactosidase">alpha-galactosidase</a> A (α-GalA). An "amenable" mutation is one that leads to <a href="/wiki/Protein_folding" title="Protein folding">misfolding</a> of the enzyme, but otherwise would not significantly impair its function.<sup id="cite_ref-EPAR_8-1" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup> </p><p>Based on an <i><a href="/wiki/In_vitro" title="In vitro">in vitro</a></i> test, Amicus Therapeutics has published a list of 269 amenable and nearly 600 non-amenable mutations. About 35 to 50% of people with Fabry have an amenable mutation.<sup id="cite_ref-EPAR_8-2" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup> </p> <div class="mw-heading mw-heading2"><h2 id="Adverse_effects">Adverse effects</h2><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=2" title="Edit section: Adverse effects"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <p>The most common side effect in <a href="/wiki/Clinical_trial" title="Clinical trial">clinical trials</a> was headache (in about 10% of people who take it). Less common side effects (between 1 and 10% of people) included unspecific symptoms such as dizziness, <a href="/wiki/Fatigue_(medical)" class="mw-redirect" title="Fatigue (medical)">fatigue</a>, and <a href="/wiki/Nausea" title="Nausea">nausea</a>, but also depression. Possible rare side effects could not be assessed because of the low number of subjects in the clinical trials in which adverse effects were measured.<sup id="cite_ref-EPAR_8-3" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup> </p> <div class="mw-heading mw-heading2"><h2 id="Interactions">Interactions</h2><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=3" title="Edit section: Interactions"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <p>When combined with <a href="/wiki/Intravenous" class="mw-redirect" title="Intravenous">intravenous</a> <a href="/wiki/Agalsidase" class="mw-redirect" title="Agalsidase">agalsidase</a> alfa or beta, which are <a href="/wiki/Recombinant_protein" class="mw-redirect" title="Recombinant protein">recombinant</a> versions of the enzyme α-GalA, migalastat increases tissue concentrations of functional α-GalA compared to agalsidase given alone up to fivefold.Migalastat is not intended to be combined with agalsidase.<sup id="cite_ref-EPAR_8-4" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup> </p><p>Migalastat does not inhibit or induce <a href="/wiki/Cytochrome_P450" title="Cytochrome P450">cytochrome P450</a> liver enzymes or <a href="/wiki/Transporter_protein" class="mw-redirect" title="Transporter protein">transporter proteins</a> and is therefore expected to have a low potential for interactions with other drugs.<sup id="cite_ref-EPAR_8-5" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup> </p> <div class="mw-heading mw-heading2"><h2 id="Pharmacology">Pharmacology</h2><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=4" title="Edit section: Pharmacology"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <div class="mw-heading mw-heading3"><h3 id="Mechanism_of_action">Mechanism of action</h3><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=5" title="Edit section: Mechanism of action"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <p>Fabry disease is a <a href="/wiki/Genetic_disorder" title="Genetic disorder">genetic disorder</a> caused by various mutations of the enzyme α-GalA, which is responsible for breaking down the <a href="/wiki/Sphingolipid" title="Sphingolipid">sphingolipid</a> <a href="/wiki/Globotriaosylceramide" title="Globotriaosylceramide">globotriaosylceramide</a> (Gb3), among other <a href="/wiki/Glycolipid" title="Glycolipid">glycolipids</a> and <a href="/wiki/Glycoprotein" title="Glycoprotein">glycoproteins</a>. Some of these mutations result in misfolding of α-GalA, which subsequently fails protein quality control in the <a href="/wiki/Endoplasmic_reticulum" title="Endoplasmic reticulum">endoplasmic reticulum</a> and is decomposed. Lack of functional α-GalA leads to accumulation of Gb3 in blood vessels and other tissues, with a wide range of symptoms including kidney, heart, and skin problems.<sup id="cite_ref-Sanches_11-0" class="reference"><a href="#cite_note-Sanches-11"><span class="cite-bracket">[</span>11<span class="cite-bracket">]</span></a></sup> </p><p>Migalastat is a potent, orally available <a href="/wiki/Enzyme_inhibitor" title="Enzyme inhibitor">inhibitor</a> of α-GalA (<a href="/wiki/Half_maximal_inhibitory_concentration" class="mw-redirect" title="Half maximal inhibitory concentration">IC<sub>50</sub></a>: 4 <a href="/wiki/Micromolar" class="mw-redirect" title="Micromolar">μM</a>).<sup id="cite_ref-Sanches_11-1" class="reference"><a href="#cite_note-Sanches-11"><span class="cite-bracket">[</span>11<span class="cite-bracket">]</span></a></sup> When binding to faulty α-GalA, it shifts the folding behaviour towards the proper conformation, resulting in a functional enzyme provided the mutation is amenable.<sup id="cite_ref-EPAR_8-6" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup> Molecules with this type of mechanism are called <a href="/wiki/Pharmacological_chaperone" title="Pharmacological chaperone">pharmacological chaperones</a>.<sup id="cite_ref-Sanches_11-2" class="reference"><a href="#cite_note-Sanches-11"><span class="cite-bracket">[</span>11<span class="cite-bracket">]</span></a></sup> </p><p>When the enzyme reaches its destination, the <a href="/wiki/Lysosome" title="Lysosome">lysosome</a>, migalastat dissociates because of the low <a href="/wiki/PH" title="PH">pH</a> and the relative abundance of Gb3 and other <a href="/wiki/Substrate_(biochemistry)" class="mw-redirect" title="Substrate (biochemistry)">substrates</a>, leaving α-GalA free to fulfill its function. Depending on the mutation, the <a href="/wiki/Half_maximal_effective_concentration" class="mw-redirect" title="Half maximal effective concentration">EC<sub>50</sub></a> is between 0.8 μM and over 1 mM in cellular models.<sup id="cite_ref-Benjamin_12-0" class="reference"><a href="#cite_note-Benjamin-12"><span class="cite-bracket">[</span>12<span class="cite-bracket">]</span></a></sup> </p> <ul class="gallery mw-gallery-traditional"> <li class="gallerybox" style="width: 155px"> <div class="thumb" style="width: 150px; height: 150px;"><span typeof="mw:File"><a href="/wiki/File:PBB_Protein_GLA_image.jpg" class="mw-file-description" title="The enzyme alpha-galactosidase A (α-GalA)"><img alt="The enzyme alpha-galactosidase A (α-GalA)" src="//upload.wikimedia.org/wikipedia/commons/thumb/3/31/PBB_Protein_GLA_image.jpg/120px-PBB_Protein_GLA_image.jpg" decoding="async" width="120" height="120" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/3/31/PBB_Protein_GLA_image.jpg/180px-PBB_Protein_GLA_image.jpg 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/3/31/PBB_Protein_GLA_image.jpg/240px-PBB_Protein_GLA_image.jpg 2x" data-file-width="500" data-file-height="500" /></a></span></div> <div class="gallerytext">The enzyme <a href="/wiki/Alpha-galactosidase" class="mw-redirect" title="Alpha-galactosidase">alpha-galactosidase</a> A (α-GalA)</div> </li> <li class="gallerybox" style="width: 155px"> <div class="thumb" style="width: 150px; height: 150px;"><span typeof="mw:File"><a href="/wiki/File:Globotriaosylceramide.svg" class="mw-file-description" title="Globotriaosylceramide (Gb3), a substrate of α-GalA, has a terminal D-galactose structurally similar to migalastat.[13]"><img alt="Globotriaosylceramide (Gb3), a substrate of α-GalA, has a terminal D-galactose structurally similar to migalastat.[13]" src="//upload.wikimedia.org/wikipedia/commons/thumb/5/5f/Globotriaosylceramide.svg/110px-Globotriaosylceramide.svg.png" decoding="async" width="110" height="120" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/5/5f/Globotriaosylceramide.svg/166px-Globotriaosylceramide.svg.png 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/5/5f/Globotriaosylceramide.svg/221px-Globotriaosylceramide.svg.png 2x" data-file-width="990" data-file-height="1075" /></a></span></div> <div class="gallerytext"><a href="/wiki/Globotriaosylceramide" title="Globotriaosylceramide">Globotriaosylceramide</a> (Gb3), a <a href="/wiki/Substrate_(biochemistry)" class="mw-redirect" title="Substrate (biochemistry)">substrate</a> of α-GalA, has a terminal <small>D</small>-<a href="/wiki/Galactose" title="Galactose">galactose</a> structurally similar to migalastat.<sup id="cite_ref-Warnock_13-0" class="reference"><a href="#cite_note-Warnock-13"><span class="cite-bracket">[</span>13<span class="cite-bracket">]</span></a></sup></div> </li> <li class="gallerybox" style="width: 155px"> <div class="thumb" style="width: 150px; height: 150px;"><span typeof="mw:File"><a href="/wiki/File:1-Deoxygalactonojirimycin_2.svg" class="mw-file-description" title="Migalastat ("top" view)"><img alt="Migalastat ("top" view)" src="//upload.wikimedia.org/wikipedia/commons/thumb/c/c5/1-Deoxygalactonojirimycin_2.svg/120px-1-Deoxygalactonojirimycin_2.svg.png" decoding="async" width="120" height="89" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/c/c5/1-Deoxygalactonojirimycin_2.svg/180px-1-Deoxygalactonojirimycin_2.svg.png 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/c/c5/1-Deoxygalactonojirimycin_2.svg/240px-1-Deoxygalactonojirimycin_2.svg.png 2x" data-file-width="512" data-file-height="378" /></a></span></div> <div class="gallerytext">Migalastat ("top" view)</div> </li> </ul> <div class="mw-heading mw-heading3"><h3 id="Pharmacokinetics">Pharmacokinetics</h3><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=6" title="Edit section: Pharmacokinetics"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <p>Migalastat is almost completely absorbed from the gut; taking the drug together with food decreases its absorption by about 40%. Total <a href="/wiki/Bioavailability" title="Bioavailability">bioavailability</a> is about 75% when taken without food. The substance is not bound to blood <a href="/wiki/Plasma_proteins" class="mw-redirect" title="Plasma proteins">plasma proteins</a>.<sup id="cite_ref-EPAR_8-7" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup> </p><p>Only a small fraction of a migalastat dose is metabolized, mainly to three <a href="/wiki/Dehydrogenated" class="mw-redirect" title="Dehydrogenated">dehydrogenated</a> <i>O</i>-<a href="/wiki/Glucuronide" title="Glucuronide">glucuronides</a> (4% of the dose) and a number of unspecified metabolites (10%). The drug is mainly eliminated via the urine (77%) and to a smaller extent via the faeces (20%). Practically all of the metabolites are excreted in the urine. <a href="/wiki/Elimination_half-life" class="mw-redirect" title="Elimination half-life">Elimination half-life</a> is three to five hours after a single dose.<sup id="cite_ref-EPAR_8-8" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup> </p> <div class="mw-heading mw-heading2"><h2 id="Chemistry">Chemistry</h2><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=7" title="Edit section: Chemistry"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <figure class="mw-default-size" typeof="mw:File/Thumb"><a href="/wiki/File:Galactose-standard.svg" class="mw-file-description"><img src="//upload.wikimedia.org/wikipedia/commons/thumb/c/cc/Galactose-standard.svg/150px-Galactose-standard.svg.png" decoding="async" width="150" height="97" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/c/cc/Galactose-standard.svg/225px-Galactose-standard.svg.png 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/c/cc/Galactose-standard.svg/300px-Galactose-standard.svg.png 2x" data-file-width="512" data-file-height="331" /></a><figcaption><small>D</small>-<a href="/wiki/Galactose" title="Galactose">Galactose</a>, for comparison</figcaption></figure> <p>Migalastat is used in form of the <a href="/wiki/Hydrochloride" title="Hydrochloride">hydrochloride</a>, which is a white crystalline solid and is soluble in water.<sup id="cite_ref-14" class="reference"><a href="#cite_note-14"><span class="cite-bracket">[</span>14<span class="cite-bracket">]</span></a></sup><sup class="reference nowrap"><span title="Page / location: 11">: 11 </span></sup> The molecule has four <a href="/wiki/Asymmetric_carbon" title="Asymmetric carbon">asymmetric carbon</a> atoms with the same <a href="/wiki/Stereochemistry" title="Stereochemistry">stereochemistry</a> as the sugar <small>D</small>-<a href="/wiki/Galactose" title="Galactose">galactose</a>, but is missing the first <a href="/wiki/Hydroxyl" class="mw-redirect" title="Hydroxyl">hydroxyl</a> group. It has a nitrogen atom in the ring instead of an oxygen, which makes it an <a href="/wiki/Iminosugar" title="Iminosugar">iminosugar</a>.<sup id="cite_ref-Asano_15-0" class="reference"><a href="#cite_note-Asano-15"><span class="cite-bracket">[</span>15<span class="cite-bracket">]</span></a></sup> </p><p>The structure is formally derived from <a href="/wiki/Nojirimycin" title="Nojirimycin">nojirimycin</a>.<sup class="noprint Inline-Template Template-Fact" style="white-space:nowrap;">[<i><a href="/wiki/Wikipedia:Citation_needed" title="Wikipedia:Citation needed"><span title="This claim needs references to reliable sources. (October 2023)">citation needed</span></a></i>]</sup> </p> <div class="mw-heading mw-heading2"><h2 id="History">History</h2><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=8" title="Edit section: History"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <p>Migalastat was isolated as a <a href="/wiki/Fermentation" title="Fermentation">fermentation</a> product of the bacterium <i><a href="/wiki/Streptomyces_lydicus" title="Streptomyces lydicus">Streptomyces lydicus</a></i> (<a href="/wiki/Strain_(biology)" title="Strain (biology)">strain</a> PA-5726) in 1988 and called <i>1-deoxygalactonojirimycin</i>.<sup id="cite_ref-Asano_15-1" class="reference"><a href="#cite_note-Asano-15"><span class="cite-bracket">[</span>15<span class="cite-bracket">]</span></a></sup><sup id="cite_ref-Miyake_16-0" class="reference"><a href="#cite_note-Miyake-16"><span class="cite-bracket">[</span>16<span class="cite-bracket">]</span></a></sup> In 2004, it was designated orphan drug status by the US FDA for the treatment of Fabry disease,<sup id="cite_ref-Orphans_5-1" class="reference"><a href="#cite_note-Orphans-5"><span class="cite-bracket">[</span>5<span class="cite-bracket">]</span></a></sup> and in 2006 the European CHMP did likewise.<sup id="cite_ref-EMA_17-0" class="reference"><a href="#cite_note-EMA-17"><span class="cite-bracket">[</span>17<span class="cite-bracket">]</span></a></sup> The sponsorship for the drug was transferred several times over the following years: from Amicus Therapeutics to <a href="/wiki/Shire_Pharmaceuticals" class="mw-redirect" title="Shire Pharmaceuticals">Shire Pharmaceuticals</a> in 2008, back to Amicus in 2010, to <a href="/wiki/Glaxo" class="mw-redirect" title="Glaxo">Glaxo</a> in 2011, and again to Amicus in 2014.<sup id="cite_ref-OrphanEU_18-0" class="reference"><a href="#cite_note-OrphanEU-18"><span class="cite-bracket">[</span>18<span class="cite-bracket">]</span></a></sup> </p><p>Two phase III clinical trials with a total of about 110 subjects were conducted between 2009 and 2015, one <a href="/wiki/Double-blind" class="mw-redirect" title="Double-blind">double-blind</a> comparing the drug to <a href="/wiki/Placebo" title="Placebo">placebo</a>, and one comparing it to recombinant α-GalA without blinding. Migalastat stabilised heart and <a href="/wiki/Kidney_function" class="mw-redirect" title="Kidney function">kidney function</a> over the 30-months period of these trials.<sup id="cite_ref-EPAR_8-9" class="reference"><a href="#cite_note-EPAR-8"><span class="cite-bracket">[</span>8<span class="cite-bracket">]</span></a></sup> </p><p>In September 2015, Amicus announced that it would submit a <a href="/wiki/New_drug_application" class="mw-redirect" title="New drug application">new drug application</a> (NDA) for <a href="/wiki/Accelerated_approval_(FDA)" title="Accelerated approval (FDA)">accelerated approval</a> of migalastat to the FDA by the end of 2015.<sup id="cite_ref-NDA_19-0" class="reference"><a href="#cite_note-NDA-19"><span class="cite-bracket">[</span>19<span class="cite-bracket">]</span></a></sup> The CHMP recommended approval in April 2016, but the FDA rejected the application in November for having insufficient data in November 2016.<sup id="cite_ref-20" class="reference"><a href="#cite_note-20"><span class="cite-bracket">[</span>20<span class="cite-bracket">]</span></a></sup> The drug was approved in the European Union in May 2016.<sup id="cite_ref-Newswire_7-1" class="reference"><a href="#cite_note-Newswire-7"><span class="cite-bracket">[</span>7<span class="cite-bracket">]</span></a></sup> Germany was the first country where migalastat was launched.<sup id="cite_ref-Newswire_7-2" class="reference"><a href="#cite_note-Newswire-7"><span class="cite-bracket">[</span>7<span class="cite-bracket">]</span></a></sup> After <a href="/wiki/Scott_Gottlieb" title="Scott Gottlieb">Scott Gottlieb</a> became FDA commissioner in 2017, the CEO of Amicus began lobbying him directly for the FDA to accept the NDA and in February 2018 the FDA accepted it and promised a response by August 2018.<sup id="cite_ref-21" class="reference"><a href="#cite_note-21"><span class="cite-bracket">[</span>21<span class="cite-bracket">]</span></a></sup> </p> <div class="mw-heading mw-heading2"><h2 id="See_also">See also</h2><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=9" title="Edit section: See also"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <ul><li><a href="/wiki/Miglustat" title="Miglustat">Miglustat</a>, a drug for the treatment of Gaucher disease, with a similar structure</li> <li><a href="/wiki/1-Deoxynojirimycin" title="1-Deoxynojirimycin">1-Deoxynojirimycin</a>, a stereoisomer of migalastat</li></ul> <div class="mw-heading mw-heading2"><h2 id="References">References</h2><span class="mw-editsection"><span class="mw-editsection-bracket">[</span><a href="/w/index.php?title=Migalastat&action=edit&section=10" title="Edit section: References"><span>edit</span></a><span class="mw-editsection-bracket">]</span></span></div> <style data-mw-deduplicate="TemplateStyles:r1239543626">.mw-parser-output .reflist{margin-bottom:0.5em;list-style-type:decimal}@media screen{.mw-parser-output .reflist{font-size:90%}}.mw-parser-output .reflist .references{font-size:100%;margin-bottom:0;list-style-type:inherit}.mw-parser-output .reflist-columns-2{column-width:30em}.mw-parser-output .reflist-columns-3{column-width:25em}.mw-parser-output .reflist-columns{margin-top:0.3em}.mw-parser-output .reflist-columns ol{margin-top:0}.mw-parser-output .reflist-columns li{page-break-inside:avoid;break-inside:avoid-column}.mw-parser-output .reflist-upper-alpha{list-style-type:upper-alpha}.mw-parser-output .reflist-upper-roman{list-style-type:upper-roman}.mw-parser-output .reflist-lower-alpha{list-style-type:lower-alpha}.mw-parser-output .reflist-lower-greek{list-style-type:lower-greek}.mw-parser-output .reflist-lower-roman{list-style-type:lower-roman}</style><div class="reflist"> <div class="mw-references-wrap mw-references-columns"><ol class="references"> <li id="cite_note-1"><span class="mw-cite-backlink"><b><a href="#cite_ref-1">^</a></b></span> <span class="reference-text"><style data-mw-deduplicate="TemplateStyles:r1238218222">.mw-parser-output cite.citation{font-style:inherit;word-wrap:break-word}.mw-parser-output .citation q{quotes:"\"""\"""'""'"}.mw-parser-output .citation:target{background-color:rgba(0,127,255,0.133)}.mw-parser-output .id-lock-free.id-lock-free a{background:url("//upload.wikimedia.org/wikipedia/commons/6/65/Lock-green.svg")right 0.1em center/9px no-repeat}.mw-parser-output .id-lock-limited.id-lock-limited a,.mw-parser-output .id-lock-registration.id-lock-registration a{background:url("//upload.wikimedia.org/wikipedia/commons/d/d6/Lock-gray-alt-2.svg")right 0.1em center/9px no-repeat}.mw-parser-output .id-lock-subscription.id-lock-subscription a{background:url("//upload.wikimedia.org/wikipedia/commons/a/aa/Lock-red-alt-2.svg")right 0.1em center/9px no-repeat}.mw-parser-output .cs1-ws-icon 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.citation .mw-selflink{font-weight:inherit}@media screen{.mw-parser-output .cs1-format{font-size:95%}html.skin-theme-clientpref-night .mw-parser-output .cs1-maint{color:#18911f}}@media screen and (prefers-color-scheme:dark){html.skin-theme-clientpref-os .mw-parser-output .cs1-maint{color:#18911f}}</style><cite class="citation web cs1"><a rel="nofollow" class="external text" href="https://www.tga.gov.au/resources/prescription-medicines-registrations/galafold-amicus-therapeutics-pty-ltd">"Galafold (Amicus Therapeutics Pty Ltd)"</a>. <i>tga.gov.au</i><span class="reference-accessdate">. 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out a short red carpet for Amicus' migalastat"</a>. <i>Endpoints</i>.</cite><span title="ctx_ver=Z39.88-2004&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.jtitle=Endpoints&rft.atitle=Once+rejected%2C+FDA+now+rolls+out+a+short+red+carpet+for+Amicus%27+migalastat&rft.date=2018-02-12&rft.aulast=Carroll&rft.aufirst=John&rft_id=https%3A%2F%2Fendpts.com%2Fonce-rejected-fda-now-rolls-out-a-short-red-carpet-for-amicus-migalastat%2F&rfr_id=info%3Asid%2Fen.wikipedia.org%3AMigalastat" class="Z3988"></span></span> </li> </ol></div></div> <style data-mw-deduplicate="TemplateStyles:r1130092004">.mw-parser-output .portal-bar{font-size:88%;font-weight:bold;display:flex;justify-content:center;align-items:baseline}.mw-parser-output .portal-bar-bordered{padding:0 2em;background-color:#fdfdfd;border:1px solid #a2a9b1;clear:both;margin:1em auto 0}.mw-parser-output .portal-bar-related{font-size:100%;justify-content:flex-start}.mw-parser-output .portal-bar-unbordered{padding:0 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