{"id":1386,"date":"2026-09-03T09:36:00","date_gmt":"2026-09-03T09:36:00","guid":{"rendered":"https:\/\/vistaralabs.ca\/ss-31-elamipretide-human-trials\/"},"modified":"2026-09-04T08:24:56","modified_gmt":"2026-09-04T08:24:56","slug":"ss-31-elamipretide-human-trials","status":"publish","type":"post","link":"https:\/\/vistaralabs.ca\/fr\/ss-31-elamipretide-human-trials\/","title":{"rendered":"SS-31 (Elamipretide): From Research to FDA Approval"},"content":{"rendered":"<p><strong>SS-31 \u2014 known in the clinic as elamipretide \u2014 is a mitochondria-targeted peptide that has reached large randomized trials.<\/strong> Its record is mixed, and reading it honestly means noting both the wins and the misses.<\/p>\n<h2>Findings<\/h2>\n<ul class=\"vl-findings\">\n<li>In late 2025, elamipretide received its <strong>first FDA approval<\/strong> \u2014 SS-31&#8217;s clinical form is now an approved medicine.<\/li>\n<li>SS-31 (called <strong>elamipretide<\/strong> in the clinic) targets the mitochondria, the cell&#8217;s power plants, and has been tested in large randomized trials.<\/li>\n<li>In Barth syndrome, patients on long-term elamipretide walked about <strong>96 metres farther<\/strong> in the standard six-minute walking test.<\/li>\n<li>In a trial for dry age-related macular degeneration, it reduced a key measure of retinal-cell loss by <strong>43%<\/strong>.<\/li>\n<li>The Barth syndrome approval is the first ever for a mitochondria-targeted therapeutic of this class.<\/li>\n<\/ul>\n<h2>From trial compound to approved medicine (2025)<\/h2>\n<p>In late 2025, elamipretide received its first FDA approval, for Barth syndrome \u2014 making it the first cardiolipin-targeted mitochondrial therapeutic ever approved (&#8220;Elamipretide: First Approval,&#8221; <em>Drugs<\/em>, 2026). For a compound that began as a research peptide, that is the full distance: laboratory, randomized trials, approval.<\/p>\n<p>The approval rests on the trial record covered below, with the Barth syndrome walking-distance gains carrying the weight. It also changes how the rest of the program reads: elamipretide is no longer a candidate molecule. It is a medicine with an expanding evidence base.<\/p>\n<h2>Barth syndrome: the clearest positive (Genet Med, 2024)<\/h2>\n<p>The TAZPOWER trial&#8217;s 168-week open-label extension followed patients with Barth syndrome on daily elamipretide 40 mg. Eight of ten patients reached the week-168 visit. There were significant improvements on the six-minute walk test at every time point, totalling a cumulative 96.1 metres of improvement (P=0.003 at week 168), along with improved fatigue scores and measurable gains in left-ventricular function. It was well tolerated, with injection-site reactions the most common adverse event.<\/p>\n<h2>Macular degeneration: positive on secondary measures (Ophthalmol Sci, 2024)<\/h2>\n<p>The Phase II ReCLAIM-2 trial in dry age-related macular degeneration did not meet its primary endpoints, but elamipretide produced a 43% reduction in progression of ellipsoid-zone loss (a marker of photoreceptor damage, P=0.0034) and significantly more patients gained at least 10 letters of low-luminance visual acuity (14.6% vs 2.1%, P=0.04).<\/p>\n<h2>Where it fell short (Neurology, 2023)<\/h2>\n<p>The pivotal Phase 3 MMPOWER-3 trial in primary mitochondrial myopathy (218 patients) did not meet its primary endpoints on the six-minute walk test or fatigue score, though treatment was well tolerated. The compound&#8217;s human story is therefore condition-specific rather than uniform.<\/p>\n<h2>What SS-31 is<\/h2>\n<p>SS-31 (elamipretide) is a mitochondria-targeted tetrapeptide that accumulates in the inner mitochondrial membrane, binding cardiolipin and studied for its effect on cellular energy. Our <a href=\"https:\/\/vistaralabs.ca\/buy-ss-31-canada\/\">SS-31 buying guide<\/a> covers the compound and its handling.<\/p>\n<div class=\"vl-note\">\n<p>Vistara Labs stocks <a href=\"https:\/\/vistaralabs.ca\/ss-31-10mg\/\">SS-31 10&nbsp;mg<\/a> research vials, shipped from within Canada to every province.<\/p>\n<\/div>\n<h2>Frequently asked questions<\/h2>\n<h3>Has SS-31 shown positive results in humans?<\/h3>\n<p>Yes, in specific settings. In a 168-week open-label extension in Barth syndrome patients, elamipretide (SS-31) produced a cumulative 96.1-metre improvement on the six-minute walk test and improvements in cardiac function. A macular-degeneration trial slowed a marker of photoreceptor loss.<\/p>\n<h3>Did every SS-31 trial succeed?<\/h3>\n<p>No, and it&#8217;s worth being precise. The large MMPOWER-3 primary mitochondrial myopathy trial did not meet its primary endpoints. The positive signals came in the Barth syndrome extension and, on secondary measures, in the eye-disease trial.<\/p>\n<h3>What is SS-31?<\/h3>\n<p>SS-31 (elamipretide) is a mitochondria-targeted tetrapeptide that concentrates in the inner mitochondrial membrane, where it is studied for its effect on cellular energy production.<\/p>\n<p class=\"vl-citation\"><em>Sources: Thompson WR, et al. Long-term efficacy and safety of elamipretide in Barth syndrome (TAZPOWER 168-week OLE). Genet Med. 2024;26:101138. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/38602181\/\" rel=\"nofollow noopener\" target=\"_blank\">PMID 38602181<\/a>. Ehlers JP, et al. ReCLAIM-2: Elamipretide in age-related macular degeneration. Ophthalmol Sci. 2024;5:100628. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/39605874\/\" rel=\"nofollow noopener\" target=\"_blank\">PMID 39605874<\/a>. Karaa A, et al. Elamipretide in primary mitochondrial myopathy (MMPOWER-3). Neurology. 2023;101:e238-e252. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/37268435\/\" rel=\"nofollow noopener\" target=\"_blank\">PMID 37268435<\/a>.<\/em><\/p>\n<p><script type=\"application\/ld+json\">{\"@context\": \"https:\/\/schema.org\", \"@type\": \"FAQPage\", \"mainEntity\": [{\"@type\": \"Question\", \"name\": \"Has SS-31 shown positive results in humans?\", \"acceptedAnswer\": {\"@type\": \"Answer\", \"text\": \"Yes, in specific settings. In a 168-week open-label extension in Barth syndrome patients, elamipretide (SS-31) produced a cumulative 96.1-metre improvement on the six-minute walk test and improvements in cardiac function. A macular-degeneration trial slowed a marker of photoreceptor loss.\"}}, {\"@type\": \"Question\", \"name\": \"Did every SS-31 trial succeed?\", \"acceptedAnswer\": {\"@type\": \"Answer\", \"text\": \"No, and it's worth being precise. The large MMPOWER-3 primary mitochondrial myopathy trial did not meet its primary endpoints. The positive signals came in the Barth syndrome extension and, on secondary measures, in the eye-disease trial.\"}}, {\"@type\": \"Question\", \"name\": \"What is SS-31?\", \"acceptedAnswer\": {\"@type\": \"Answer\", \"text\": \"SS-31 (elamipretide) is a mitochondria-targeted tetrapeptide that concentrates in the inner mitochondrial membrane, where it is studied for its effect on cellular energy production.\"}}]}<\/script><\/p>\n","protected":false},"excerpt":{"rendered":"<p>SS-31 (elamipretide) produced a 96-metre walk-test improvement in Barth syndrome and slowed photoreceptor loss in eye disease, while missing endpoints in mitochondrial myopathy. The complete human record.<\/p>","protected":false},"author":1,"featured_media":1282,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"_kad_post_transparent":"","_kad_post_title":"","_kad_post_layout":"","_kad_post_sidebar_id":"","_kad_post_content_style":"","_kad_post_vertical_padding":"","_kad_post_feature":"","_kad_post_feature_position":"","_kad_post_header":false,"_kad_post_footer":false,"_kad_post_classname":"","_vistara_fr_title":"","_vistara_fr_desc":"","_vl_research_product":1131,"footnotes":""},"categories":[33],"tags":[],"vl_research_area":[42],"class_list":["post-1386","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-peptide-research"],"_links":{"self":[{"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/posts\/1386","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/comments?post=1386"}],"version-history":[{"count":3,"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/posts\/1386\/revisions"}],"predecessor-version":[{"id":1457,"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/posts\/1386\/revisions\/1457"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/media\/1282"}],"wp:attachment":[{"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/media?parent=1386"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/categories?post=1386"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/tags?post=1386"},{"taxonomy":"vl_research_area","embeddable":true,"href":"https:\/\/vistaralabs.ca\/fr\/wp-json\/wp\/v2\/vl_research_area?post=1386"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}