{"id":132,"date":"2026-09-22T13:06:09","date_gmt":"2026-09-22T13:06:09","guid":{"rendered":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/?page_id=132"},"modified":"2026-10-30T10:53:26","modified_gmt":"2026-10-30T10:53:26","slug":"pipeline","status":"publish","type":"page","link":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/pipeline\/","title":{"rendered":"Pipeline"},"content":{"rendered":"<main id='oxy-main'><div class=\"oxy-container-132-269 oxy-container od-page-pipeline\"><div class=\"oxy-container-132-268 oxy-container od-fx-793346c73\"><section class=\"oxy-container-132-282 oxy-container od-hero\" aria-labelledby=\"pipeline-title\"><div class=\"oxy-container-132-281 oxy-container od-container\"><div class=\"oxy-container-132-272 oxy-container od-hero-heading\"><div class=\"oxy-svg-icon2-132-270 oxy-svg-icon2 od-hero-icon\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"24\" height=\"24\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"M14 2v6a2 2 0 0 0 .245.96l5.51 10.08A2 2 0 0 1 18 22H6a2 2 0 0 1-1.755-2.96l5.51-10.08A2 2 0 0 0 10 8V2\"><\/path><path d=\"M6.453 15h11.094\"><\/path><path d=\"M8.5 2h7\"><\/path><\/svg>\n<\/div><h1 class=\"oxy-text-132-271 oxy-text od-hero-title\" id=\"pipeline-title\">\nDevelopment Pipeline\n<\/h1><\/div><p class=\"oxy-text-132-273 oxy-text od-hero-lead\">\nOur commitment to advancing rare disease treatments through innovative research and development\n<\/p><p class=\"oxy-text-132-274 oxy-text od-hero-copy\">\nExplore our pipeline of therapies in development, from early-stage research to regulatory filing.\n<\/p><div class=\"oxy-container-132-277 oxy-container od-hero-actions\"><a class=\"oxy-text-link-132-275 oxy-text-link od-button od-button--accent od-button--borderless\" href=\"\/products\/\" target=\"_self\">\nExplore Our Products\n<\/a><a class=\"oxy-text-link-132-276 oxy-text-link od-button od-button--outlined-light\" href=\"\/iir\/\" target=\"_self\">\nResearch Collaborations\n<\/a><\/div><button class=\"oxy-container-link-132-280 oxy-container-link od-hero-back od-pipeline-back\" target=\"_self\" data-interactions=\"[{&quot;trigger&quot;:&quot;click&quot;,&quot;prevent_default&quot;:true,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanHeroBack&quot;}]}]\" id=\"pipeline-back\" type=\"button\" aria-label=\"Go back to the previous page\"><div class=\"oxy-svg-icon2-132-278 oxy-svg-icon2 od-hero-back-icon\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"24\" height=\"24\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"m15 18-6-6 6-6\"><\/path><\/svg>\n<\/div><span class=\"oxy-text-132-279 oxy-text od-link-label\">\nBack\n<\/span><\/button><\/div><\/section><section class=\"oxy-container-132-289 oxy-container od-section\" aria-labelledby=\"pipeline-advancing-title\"><div class=\"oxy-container-132-288 oxy-container od-container\"><header class=\"oxy-container-132-287 oxy-container od-pipeline-intro\"><span class=\"oxy-text-132-283 oxy-text od-eyebrow\">\nWE INNOVATE\n<\/span><h2 class=\"oxy-text-132-284 oxy-text od-home-heading-title od-pipeline-intro-title\" id=\"pipeline-advancing-title\">\nAdvancing Treatment Standards for Rare Diseases\n<\/h2><p class=\"oxy-text-132-285 oxy-text od-pipeline-intro-lead\">\nOrphalan's pipeline is focused on unmet needs of patients with rare diseases.\n<\/p><p class=\"oxy-text-132-286 oxy-text od-pipeline-intro-note\">\n<strong>Note:<\/strong> The Orphalan Pipeline data is updated quarterly.\n<\/p><\/header><\/div><\/section><section class=\"oxy-container-132-299 oxy-container od-section od-pipeline-research\" aria-labelledby=\"pipeline-driven-title\"><div class=\"oxy-container-132-298 oxy-container od-container\"><div class=\"oxy-container-132-297 oxy-container od-pipeline-research-grid\"><div class=\"oxy-container-132-294 oxy-container od-pipeline-research-copy\"><div class=\"oxy-container-132-290 oxy-container od-section-marker\" aria-hidden=\"true\"><\/div><h2 class=\"oxy-text-132-291 oxy-text od-section-title\" id=\"pipeline-driven-title\">\nDriven by Patients, Guided by Science\n<\/h2><p class=\"oxy-text-132-292 oxy-text od-body od-pipeline-research-first\">\nRare diseases often have limited treatment options available. Orphalan focuses on developing medicines with the potential to improve outcomes for patients living with these conditions.\n<\/p><p class=\"oxy-text-132-293 oxy-text od-body\">\nOur R&amp;D pipeline spans clinical-stage projects, from early-phase studies through to regulatory submission, with a focus on difficult-to-treat conditions. Some of these are new molecular entities, while others are existing products being studied for potential new indications or different formulations.\n<\/p><\/div><div class=\"oxy-container-132-296 oxy-container od-pipeline-research-media\"><img decoding=\"async\" class=\"oxy-image-132-295 oxy-image od-pipeline-research-image\" src=\"https:\/\/funny-fermi.104-248-174-66.plesk.page\/wp-content\/uploads\/2026\/09\/ORPH_PRESS_001_EDIT__1_.jpg\" alt=\"Child sitting on adult&#039;s shoulders\" loading=\"lazy\"><\/div><\/div><\/div><\/section><section class=\"oxy-container-132-400 oxy-container od-section od-pipeline-overview\" data-interactions=\"[{&quot;trigger&quot;:&quot;page_loaded&quot;,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanPipelineInit&quot;}]}]\" aria-labelledby=\"pipeline-overview-title\"><div class=\"oxy-container-132-399 oxy-container od-container\"><div class=\"oxy-container-132-300 oxy-container od-section-marker\" aria-hidden=\"true\"><\/div><h2 class=\"oxy-text-132-301 oxy-text od-section-title od-pipeline-title--compact\" id=\"pipeline-overview-title\">\nTherapeutic Pipeline\n<\/h2><p class=\"oxy-text-132-302 oxy-text od-body od-pipeline-table-intro\">\nThe table below reflects our current clinical development portfolio, spanning regulatory submissions, late-stage and early-stage programmes.\n<\/p><div class=\"oxy-container-132-379 oxy-container od-pipeline-scroll\" role=\"region\" aria-label=\"Therapeutic pipeline table; scroll horizontally on smaller screens\" tabindex=\"0\"><div class=\"oxy-container-132-378 oxy-container od-pipeline-table\" role=\"table\" aria-label=\"Clinical development portfolio\"><div class=\"oxy-container-132-312 oxy-container od-pipeline-table-header\" role=\"row\"><span class=\"oxy-text-132-303 oxy-text od-pipeline-column-title\" role=\"columnheader\">\nActive ingredient\n<\/span><span class=\"oxy-text-132-304 oxy-text od-pipeline-column-title\" role=\"columnheader\">\nPharmaceutical form\n<\/span><span class=\"oxy-text-132-305 oxy-text od-pipeline-column-title\" role=\"columnheader\">\nDosing\n<\/span><span class=\"oxy-text-132-306 oxy-text od-pipeline-column-title\" role=\"columnheader\">\nIndication\n<\/span><div class=\"oxy-container-132-310 oxy-container od-pipeline-phase-header\" role=\"columnheader\"><span class=\"oxy-text-132-307 oxy-text od-pipeline-column-title\">\nPhase\n<\/span><button class=\"oxy-container-link-132-309 oxy-container-link od-pipeline-info\" target=\"_self\" data-interactions=\"[{&quot;trigger&quot;:&quot;click&quot;,&quot;prevent_default&quot;:true,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanPipelineToggleTooltip&quot;}]},{&quot;trigger&quot;:&quot;mouse_enter&quot;,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanPipelineShowTooltip&quot;}]},{&quot;trigger&quot;:&quot;mouse_leave&quot;,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanPipelineHideTooltip&quot;}]}]\" id=\"pipeline-phase-info\" type=\"button\" aria-label=\"Phase definitions\" aria-describedby=\"pipeline-phase-tooltip\" aria-expanded=\"false\"><div class=\"oxy-svg-icon2-132-308 oxy-svg-icon2 od-pipeline-info-icon\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"14\" height=\"14\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><circle cx=\"12\" cy=\"12\" r=\"10\"><\/circle><path d=\"M12 16v-4\"><\/path><path d=\"M12 8h.01\"><\/path><\/svg>\n<\/div><\/button><\/div><span class=\"oxy-text-132-311 oxy-text od-pipeline-column-title\" role=\"columnheader\" aria-label=\"Regional details\">\n\n<\/span><\/div><div class=\"oxy-container-132-377 oxy-container od-pipeline-rows\"><article class=\"oxy-container-132-358 oxy-container od-pipeline-row\" id=\"pipeline-0\" role=\"rowgroup\" aria-label=\"Temozolomide\"><div class=\"oxy-container-132-331 oxy-container od-pipeline-table-row\" role=\"row\"><span class=\"oxy-text-132-313 oxy-text od-pipeline-ingredient\" role=\"cell\">\nTemozolomide\n<\/span><span class=\"oxy-text-132-314 oxy-text od-pipeline-form\" role=\"cell\">\nOral suspension, 40mg\/ml\n<\/span><div class=\"oxy-container-132-316 oxy-container od-pipeline-cell\" role=\"cell\"><span class=\"oxy-text-132-315 oxy-text od-pipeline-dose\">\nQD\n<\/span><\/div><div class=\"oxy-container-132-318 oxy-container od-pipeline-cell\" role=\"cell\"><a class=\"oxy-text-link-132-317 oxy-text-link od-pipeline-indication\" href=\"\/disease-info\/glioblastoma\/\" target=\"_self\">\nGlioblastoma\n<\/a><\/div><div class=\"oxy-container-132-327 oxy-container od-pipeline-phase-cell\" role=\"cell\"><div class=\"oxy-container-132-326 oxy-container od-pipeline-stepper\" role=\"img\" aria-label=\"EU: regulatory submission completed. US: planned submission, December 2026; Phase 3 completed.\"><span class=\"oxy-text-132-319 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n1\n<\/span><span class=\"oxy-text-132-320 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-321 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n2\n<\/span><span class=\"oxy-text-132-322 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-323 oxy-text od-pipeline-step od-pipeline-step--purple\" aria-hidden=\"true\">\n3\n<\/span><span class=\"oxy-text-132-324 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-325 oxy-text od-pipeline-step od-pipeline-step--teal\" aria-hidden=\"true\">\nR\n<\/span><\/div><\/div><div class=\"oxy-container-132-330 oxy-container od-pipeline-phase-cell\" role=\"cell\"><button class=\"oxy-container-link-132-329 oxy-container-link od-pipeline-expand\" target=\"_self\" data-interactions=\"[{&quot;trigger&quot;:&quot;click&quot;,&quot;prevent_default&quot;:true,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanPipelineToggleRegional&quot;}]}]\" id=\"pipeline-regional-toggle\" type=\"button\" aria-expanded=\"false\" aria-controls=\"pipeline-regional-detail\" aria-label=\"Expand regional detail\"><div class=\"oxy-svg-icon2-132-328 oxy-svg-icon2 od-pipeline-expand-icon\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"13\" height=\"13\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"m6 9 6 6 6-6\"><\/path><\/svg>\n<\/div><\/button><\/div><\/div><div class=\"oxy-container-132-357 oxy-container od-pipeline-regional\" id=\"pipeline-regional-detail\" role=\"row\" hidden=\"\"><div class=\"oxy-container-132-356 oxy-container od-pipeline-regional-panel\" role=\"cell\" aria-colspan=\"6\"><p class=\"oxy-text-132-332 oxy-text od-pipeline-regional-heading\">\nRegional development status\n<\/p><div class=\"oxy-container-132-355 oxy-container od-pipeline-regional-list\"><div class=\"oxy-container-132-343 oxy-container od-pipeline-regional-row\"><span class=\"oxy-text-132-333 oxy-text od-pipeline-region\">\nEU\n<\/span><div class=\"oxy-container-132-341 oxy-container od-pipeline-stepper\" role=\"img\" aria-label=\"EU: regulatory submission completed\"><span class=\"oxy-text-132-334 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n1\n<\/span><span class=\"oxy-text-132-335 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-336 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n2\n<\/span><span class=\"oxy-text-132-337 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-338 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n3\n<\/span><span class=\"oxy-text-132-339 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-340 oxy-text od-pipeline-step od-pipeline-step--teal\" aria-hidden=\"true\">\nR\n<\/span><\/div><span class=\"oxy-text-132-342 oxy-text od-pipeline-regional-status\">\nFiled\n<\/span><\/div><div class=\"oxy-container-132-354 oxy-container od-pipeline-regional-row\"><span class=\"oxy-text-132-344 oxy-text od-pipeline-region\">\nUS\n<\/span><div class=\"oxy-container-132-352 oxy-container od-pipeline-stepper\" role=\"img\" aria-label=\"US: planned regulatory submission, December 2026\"><span class=\"oxy-text-132-345 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n1\n<\/span><span class=\"oxy-text-132-346 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-347 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n2\n<\/span><span class=\"oxy-text-132-348 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-349 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n3\n<\/span><span class=\"oxy-text-132-350 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-351 oxy-text od-pipeline-step od-pipeline-step--pending\" aria-hidden=\"true\">\nR\n<\/span><\/div><span class=\"oxy-text-132-353 oxy-text od-pipeline-regional-status\">\nPlanned submission \u00b7 Dec 2026\n<\/span><\/div><\/div><\/div><\/div><\/article><article class=\"oxy-container-132-376 oxy-container od-pipeline-row\" id=\"pipeline-1\" role=\"rowgroup\" aria-label=\"Trientine tetrahydrochloride\"><div class=\"oxy-container-132-375 oxy-container od-pipeline-table-row\" role=\"row\"><span class=\"oxy-text-132-359 oxy-text od-pipeline-ingredient\" role=\"cell\">\nTrientine tetrahydrochloride\n<\/span><span class=\"oxy-text-132-360 oxy-text od-pipeline-form\" role=\"cell\">\nFilm-coated tablet\n<\/span><div class=\"oxy-container-132-362 oxy-container od-pipeline-cell\" role=\"cell\"><span class=\"oxy-text-132-361 oxy-text od-pipeline-dose od-pipeline-dose--once\">\nQD\n<\/span><\/div><div class=\"oxy-container-132-364 oxy-container od-pipeline-cell\" role=\"cell\"><a class=\"oxy-text-link-132-363 oxy-text-link od-pipeline-indication\" href=\"\/disease-info\/wilsons-disease\/\" target=\"_self\">\nWilson's disease\n<\/a><\/div><div class=\"oxy-container-132-373 oxy-container od-pipeline-phase-cell\" role=\"cell\"><div class=\"oxy-container-132-372 oxy-container od-pipeline-stepper\" role=\"img\" aria-label=\"Phase 2\"><span class=\"oxy-text-132-365 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n1\n<\/span><span class=\"oxy-text-132-366 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-367 oxy-text od-pipeline-step od-pipeline-step--teal\" aria-hidden=\"true\">\n2\n<\/span><span class=\"oxy-text-132-368 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-369 oxy-text od-pipeline-step\" aria-hidden=\"true\">\n3\n<\/span><span class=\"oxy-text-132-370 oxy-text od-pipeline-connector\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-371 oxy-text od-pipeline-step\" aria-hidden=\"true\">\nR\n<\/span><\/div><\/div><div class=\"oxy-container-132-374 oxy-container od-pipeline-phase-cell\" role=\"cell\"><\/div><\/div><\/article><\/div><\/div><\/div><p class=\"oxy-container-132-383 oxy-container od-pipeline-products-note\"><span class=\"oxy-text-132-380 oxy-text od-link-label\">\nProducts that have completed development are listed on the \n<\/span><a class=\"oxy-text-link-132-381 oxy-text-link od-pipeline-inline-link\" href=\"\/products\/\" target=\"_self\">\nProducts page\n<\/a><span class=\"oxy-text-132-382 oxy-text od-link-label\">\n.\n<\/span><\/p><aside class=\"oxy-container-132-398 oxy-container od-pipeline-legend\" aria-labelledby=\"pipeline-dosing-title\"><h3 class=\"oxy-text-132-384 oxy-text od-pipeline-legend-title\" id=\"pipeline-dosing-title\">\nDosing Frequency\n<\/h3><div class=\"oxy-container-132-394 oxy-container od-pipeline-legend-items\"><div class=\"oxy-container-132-387 oxy-container od-pipeline-legend-item\"><span class=\"oxy-text-132-385 oxy-text od-pipeline-dose od-pipeline-dose--legend od-pipeline-dose--once\">\nQD\n<\/span><span class=\"oxy-text-132-386 oxy-text od-pipeline-legend-label\">\nOnce daily\n<\/span><\/div><div class=\"oxy-container-132-390 oxy-container od-pipeline-legend-item\"><span class=\"oxy-text-132-388 oxy-text od-pipeline-dose od-pipeline-dose--legend\">\nBID\n<\/span><span class=\"oxy-text-132-389 oxy-text od-pipeline-legend-label\">\nTwice daily\n<\/span><\/div><div class=\"oxy-container-132-393 oxy-container od-pipeline-legend-item\"><span class=\"oxy-text-132-391 oxy-text od-pipeline-dose od-pipeline-dose--legend\">\nBID\u2011QID\n<\/span><span class=\"oxy-text-132-392 oxy-text od-pipeline-legend-label\">\n2\u20134 times a day\n<\/span><\/div><\/div><div class=\"oxy-container-132-397 oxy-container od-pipeline-legend-footer\"><span class=\"oxy-text-132-395 oxy-text od-pipeline-updated\">\nPipeline data updated quarterly.\n<\/span><span class=\"oxy-text-132-396 oxy-text od-pipeline-last-updated\">\nLast updated: September 2026\n<\/span><\/div><\/aside><\/div><\/section><section class=\"oxy-container-132-409 oxy-container od-pipeline-registry\" aria-labelledby=\"pipeline-registry-title\"><div class=\"oxy-container-132-408 oxy-container od-container od-pipeline-narrow\"><div class=\"oxy-container-132-407 oxy-container od-pipeline-registry-layout\"><div class=\"oxy-container-132-403 oxy-container od-pipeline-registry-copy\"><h2 class=\"oxy-text-132-401 oxy-text od-pipeline-registry-title\" id=\"pipeline-registry-title\">\niWilson's Registry\n<\/h2><p class=\"oxy-text-132-402 oxy-text od-pipeline-registry-body\">\nWilson's disease data is also collected via our iWilson's Disease Registry.\n<\/p><\/div><a class=\"oxy-container-link-132-406 oxy-container-link od-button od-home-control od-home-control--teal od-pipeline-registry-action\" href=\"\/iwilsons-registry\/\" target=\"_self\"><span class=\"oxy-text-132-404 oxy-text od-link-label\">\nVisit the Registry\n<\/span><div class=\"oxy-svg-icon2-132-405 oxy-svg-icon2 od-link-icon\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"24\" height=\"24\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"M5 12h14\"><\/path><path d=\"m12 5 7 7-7 7\"><\/path><\/svg>\n<\/div><\/a><\/div><\/div><\/section><section class=\"oxy-container-132-447 oxy-container od-section od-pipeline-diagnostics\" aria-labelledby=\"pipeline-diagnostic-title\"><div class=\"oxy-container-132-446 oxy-container od-container\"><div class=\"oxy-container-132-410 oxy-container od-section-marker\" aria-hidden=\"true\"><\/div><h2 class=\"oxy-text-132-411 oxy-text od-section-title od-pipeline-title--compact\" id=\"pipeline-diagnostic-title\">\nDiagnostic Innovation\n<\/h2><p class=\"oxy-text-132-412 oxy-text od-body od-pipeline-diagnostic-lead\">\nBeyond treatments, we are developing new diagnostic tools to help doctors detect and monitor rare diseases more accurately.\n<\/p><article class=\"oxy-container-132-445 oxy-container od-pipeline-diagnostic-card\"><div class=\"oxy-container-132-444 oxy-container od-pipeline-diagnostic-grid\"><div class=\"oxy-container-132-422 oxy-container od-pipeline-diagnostic-copy\"><span class=\"oxy-text-132-413 oxy-text od-pipeline-available\">\nAvailable in the US since April 2025\n<\/span><h3 class=\"oxy-text-132-414 oxy-text od-card-title\">\nNCC Assay: Measuring Non-Ceruloplasmin Copper\n<\/h3><p class=\"oxy-text-132-415 oxy-text od-body od-pipeline-diagnostic-body\">\nCopper is an essential dietary element, usually bound tightly in blood to a protein called ceruloplasmin. In Wilson's disease, the inability to remove excess copper leads to accumulation of \"free\" copper (non-ceruloplasmin copper, or NCC) in the blood. This excess NCC is directly responsible for the organ damage seen in Wilson's disease.\n<\/p><p class=\"oxy-text-132-416 oxy-text od-body od-pipeline-diagnostic-body\">\nCritically, there are no validated tests readily available to measure NCC, making it difficult for physicians to monitor copper levels and assess treatment response. As part of the pivotal Phase 3 CHELATE trial, Orphalan was asked by the FDA to develop a new assay for NCC. This reliable and reproducible test has been validated and is now being made widely available to healthcare professionals.\n<\/p><p class=\"oxy-text-132-417 oxy-text od-body od-pipeline-diagnostic-body od-pipeline-diagnostic-body--last\">\nThe NCC assay was made available to healthcare professionals in the United States in April 2025, providing a valuable new tool for monitoring Wilson's disease treatment effectiveness.\n<\/p><a class=\"oxy-container-link-132-421 oxy-container-link od-pipeline-research-link\" href=\"https:\/\/www.thelancet.com\/journals\/langas\/article\/PIIS2468-1253(22)00284-9\/abstract\" target=\"_blank\" data-interactions=\"[{&quot;trigger&quot;:&quot;click&quot;,&quot;prevent_default&quot;:true,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanPipelineOpenExternal&quot;}]}]\" id=\"pipeline-research-link\" rel=\"noopener noreferrer\"><div class=\"oxy-svg-icon2-132-418 oxy-svg-icon2 od-link-icon\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"24\" height=\"24\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"M15 2H6a2 2 0 0 0-2 2v16a2 2 0 0 0 2 2h12a2 2 0 0 0 2-2V7Z\"><\/path><path d=\"M14 2v4a2 2 0 0 0 2 2h4\"><\/path><path d=\"M10 9H8\"><\/path><path d=\"M16 13H8\"><\/path><path d=\"M16 17H8\"><\/path><\/svg>\n<\/div><span class=\"oxy-text-132-419 oxy-text od-link-label\">\nRead the published research: The Lancet Gastroenterology &amp; Hepatology\n<\/span><div class=\"oxy-svg-icon2-132-420 oxy-svg-icon2 od-link-icon\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"24\" height=\"24\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"m9 18 6-6-6-6\"><\/path><\/svg>\n<\/div><\/a><\/div><dl class=\"oxy-container-132-443 oxy-container od-pipeline-detail-list\"><div class=\"oxy-container-132-427 oxy-container od-pipeline-detail\"><dt class=\"oxy-container-132-425 oxy-container od-pipeline-detail-heading\"><span class=\"oxy-text-132-423 oxy-text od-pipeline-detail-marker\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-424 oxy-text od-pipeline-detail-label\">\nDisease Area\n<\/span><\/dt><dd class=\"oxy-text-132-426 oxy-text od-pipeline-detail-value\">\nWilson's Disease\n<\/dd><\/div><div class=\"oxy-container-132-432 oxy-container od-pipeline-detail\"><dt class=\"oxy-container-132-430 oxy-container od-pipeline-detail-heading\"><span class=\"oxy-text-132-428 oxy-text od-pipeline-detail-marker od-pipeline-detail-marker--purple\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-429 oxy-text od-pipeline-detail-label\">\nInnovation Type\n<\/span><\/dt><dd class=\"oxy-text-132-431 oxy-text od-pipeline-detail-value\">\nDiagnostic Assay\n<\/dd><\/div><div class=\"oxy-container-132-437 oxy-container od-pipeline-detail\"><dt class=\"oxy-container-132-435 oxy-container od-pipeline-detail-heading\"><span class=\"oxy-text-132-433 oxy-text od-pipeline-detail-marker\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-434 oxy-text od-pipeline-detail-label\">\nRegulatory Context\n<\/span><\/dt><dd class=\"oxy-text-132-436 oxy-text od-pipeline-detail-value\">\nFDA-requested as part of CHELATE Phase 3 trial\n<\/dd><\/div><div class=\"oxy-container-132-442 oxy-container od-pipeline-detail\"><dt class=\"oxy-container-132-440 oxy-container od-pipeline-detail-heading\"><span class=\"oxy-text-132-438 oxy-text od-pipeline-detail-marker od-pipeline-detail-marker--purple\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-439 oxy-text od-pipeline-detail-label\">\nAvailability\n<\/span><\/dt><dd class=\"oxy-text-132-441 oxy-text od-pipeline-detail-value\">\nUnited States, April 2025\n<\/dd><\/div><\/dl><\/div><\/article><\/div><\/section><section class=\"oxy-container-132-456 oxy-container od-section od-pipeline-partnering\" aria-labelledby=\"pipeline-partnering-title\"><div class=\"oxy-container-132-455 oxy-container od-container\"><div class=\"oxy-container-132-454 oxy-container od-pipeline-partnering-copy\"><div class=\"oxy-container-132-448 oxy-container od-section-marker od-marker--center\" aria-hidden=\"true\"><\/div><h2 class=\"oxy-text-132-449 oxy-text od-pipeline-partnering-title\" id=\"pipeline-partnering-title\">\nInterested in collaborating on rare disease research?\n<\/h2><p class=\"oxy-text-132-450 oxy-text od-body od-pipeline-partnering-body\">\nWe welcome partnerships with researchers, clinicians and institutions committed to advancing treatments for patients with rare diseases.\n<\/p><a class=\"oxy-container-link-132-453 oxy-container-link od-pipeline-partnering-action\" href=\"\/partnering\/\" target=\"_self\"><span class=\"oxy-text-132-451 oxy-text od-link-label\">\nVisit our Partnering page\n<\/span><div class=\"oxy-svg-icon2-132-452 oxy-svg-icon2 od-link-icon\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"24\" height=\"24\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"M5 12h14\"><\/path><path d=\"m12 5 7 7-7 7\"><\/path><\/svg>\n<\/div><\/a><\/div><\/div><\/section><aside class=\"oxy-container-132-464 oxy-container od-approval-strip\" aria-label=\"Content approval information\"><div class=\"oxy-container-132-463 oxy-container od-container\"><div class=\"oxy-container-132-462 oxy-container od-approval-layout\"><div class=\"oxy-container-132-460 oxy-container od-approval-code\"><span class=\"oxy-text-132-457 oxy-text od-approval-text\">\nMLR Approval Code: ORP-EU-2026-001\n<\/span><span class=\"oxy-text-132-458 oxy-text od-approval-text\" aria-hidden=\"true\">\n\u2022\n<\/span><span class=\"oxy-text-132-459 oxy-text od-approval-text\">\nApproved: October 2026\n<\/span><\/div><p class=\"oxy-text-132-461 oxy-text od-approval-text\">\nFor Healthcare Professionals and Patients in Europe and regions where Orphalan operates\n<\/p><\/div><\/div><\/aside><aside class=\"oxy-container-132-477 oxy-container od-pipeline-tooltip\" id=\"pipeline-phase-tooltip\" role=\"tooltip\" hidden=\"\"><div class=\"oxy-container-132-469 oxy-container od-pipeline-tooltip-definitions\"><p class=\"oxy-text-132-465 oxy-text od-pipeline-tooltip-definition\">\n<strong class=\"od-pipeline-tooltip-key\">1<\/strong> - Safety and dosage studies in healthy volunteers or patients\n<\/p><p class=\"oxy-text-132-466 oxy-text od-pipeline-tooltip-definition\">\n<strong class=\"od-pipeline-tooltip-key\">2<\/strong> - Efficacy and side effect studies in patients\n<\/p><p class=\"oxy-text-132-467 oxy-text od-pipeline-tooltip-definition\">\n<strong class=\"od-pipeline-tooltip-key\">3<\/strong> - Large-scale efficacy and safety confirmation\n<\/p><p class=\"oxy-text-132-468 oxy-text od-pipeline-tooltip-definition\">\n<strong class=\"od-pipeline-tooltip-key\">R<\/strong> - Regulatory submission completed\n<\/p><\/div><div class=\"oxy-container-132-476 oxy-container od-pipeline-tooltip-regions\"><p class=\"oxy-container-132-472 oxy-container od-pipeline-tooltip-region\"><span class=\"oxy-text-132-470 oxy-text od-pipeline-region-dot\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-471 oxy-text od-pipeline-tooltip-region-label\">\nEU filing \/ approval\n<\/span><\/p><p class=\"oxy-container-132-475 oxy-container od-pipeline-tooltip-region\"><span class=\"oxy-text-132-473 oxy-text od-pipeline-region-dot od-pipeline-region-dot--purple\" aria-hidden=\"true\">\n\n<\/span><span class=\"oxy-text-132-474 oxy-text od-pipeline-tooltip-region-label\">\nUS filing \/ approval\n<\/span><\/p><\/div><\/aside><dialog class=\"oxy-container-132-487 oxy-container od-leader-dialog od-leader-external\" id=\"pipeline-external\" aria-labelledby=\"pipeline-external-title\" tabindex=\"-1\"><div class=\"oxy-container-132-479 oxy-container od-leader-external-icon\"><div class=\"oxy-svg-icon2-132-478 oxy-svg-icon2 od-leader-value-symbol\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"24\" height=\"24\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"M15 3h6v6\"><\/path><path d=\"M10 14 21 3\"><\/path><path d=\"M18 13v6a2 2 0 0 1-2 2H5a2 2 0 0 1-2-2V8a2 2 0 0 1 2-2h6\"><\/path><\/svg>\n<\/div><\/div><h2 class=\"oxy-text-132-480 oxy-text od-leader-external-title\" id=\"pipeline-external-title\">\nYou are leaving orphalan.com\n<\/h2><p class=\"oxy-text-132-481 oxy-text od-body od-leader-external-body\">\nYou are about to visit <strong>The Lancet Gastroenterology &amp; Hepatology<\/strong>, an external website. Orphalan is not responsible for the content of external sites.\n<\/p><div class=\"oxy-container-132-484 oxy-container od-pipeline-external-actions\"><a class=\"oxy-text-link-132-482 oxy-text-link od-leader-external-visit od-pipeline-external-action\" href=\"https:\/\/www.thelancet.com\/journals\/langas\/article\/PIIS2468-1253(22)00284-9\/abstract\" target=\"_blank\" data-interactions=\"[{&quot;trigger&quot;:&quot;click&quot;,&quot;prevent_default&quot;:true,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanPipelineVisit&quot;}]}]\" id=\"pipeline-external-visit\" rel=\"noopener noreferrer\">\nVisit Website\n<\/a><button class=\"oxy-text-link-132-483 oxy-text-link od-leader-external-stay od-pipeline-external-action\" target=\"_self\" data-interactions=\"[{&quot;trigger&quot;:&quot;click&quot;,&quot;prevent_default&quot;:true,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanPipelineCloseExternal&quot;}]}]\" id=\"pipeline-external-stay\" type=\"button\">\nStay on orphalan.com\n<\/button><\/div><button class=\"oxy-container-link-132-486 oxy-container-link od-leader-close od-leader-close--external\" target=\"_self\" data-interactions=\"[{&quot;trigger&quot;:&quot;click&quot;,&quot;prevent_default&quot;:true,&quot;actions&quot;:[{&quot;name&quot;:&quot;javascript_function&quot;,&quot;js_function_name&quot;:&quot;orphalanPipelineCloseExternal&quot;}]}]\" id=\"pipeline-external-close\" type=\"button\" aria-label=\"Close external site notice\"><div class=\"oxy-svg-icon2-132-485 oxy-svg-icon2 od-leader-close-icon\">\n<svg aria-hidden=\"true\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"24\" height=\"24\" viewBox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"M18 6 6 18\"><\/path><path d=\"m6 6 12 12\"><\/path><\/svg>\n<\/div><\/button><\/dialog><\/div><\/div><\/main>","protected":false},"excerpt":{"rendered":"<p>Development Pipeline Our commitment to advancing rare disease treatments through innovative research and development Explore our pipeline of therapies in development, from early-stage research to regulatory filing. Explore Our Products Research Collaborations Back WE INNOVATE Advancing Treatment Standards for Rare Diseases Orphalan&#8217;s pipeline is focused on unmet needs of patients with rare diseases. Note: The [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_oxygen_hide_in_design_set":false,"_oxygen_tags":"","footnotes":""},"class_list":["post-132","page","type-page","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/pages\/132","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/comments?post=132"}],"version-history":[{"count":27,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/pages\/132\/revisions"}],"predecessor-version":[{"id":1002,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/pages\/132\/revisions\/1002"}],"wp:attachment":[{"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/media?parent=132"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}