{"id":68431,"date":"2026-06-17T21:51:34","date_gmt":"2026-06-17T13:51:34","guid":{"rendered":"https:\/\/flcube.com\/?p=68431"},"modified":"2026-06-17T21:51:35","modified_gmt":"2026-06-17T13:51:35","slug":"gsks-momelotinib-receives-orphan-drug-designation-from-fda-and-ema-for-vexas-syndrome-addressing-critical-unmet-need-in-rare-myeloid-disorder","status":"publish","type":"post","link":"https:\/\/flcube.com\/?p=68431","title":{"rendered":"GSK’s Momelotinib Receives Orphan Drug Designation from FDA and EMA for VEXAS Syndrome, Addressing Critical Unmet Need in Rare Myeloid Disorder"},"content":{"rendered":"\n

GlaxoSmithKline plc (GSK)<\/strong> (NYSE: GSK<\/a>) announced that momelotinib<\/strong> has received Orphan Drug Designation (ODD)<\/strong> from both the US Food and Drug Administration (FDA)<\/strong> and European Medicines Agency (EMA)<\/strong> for the treatment of VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) syndrome<\/strong>. The rare clonal myeloid disorder, characterized by a 30-40% five-year mortality rate and no approved therapies, represents a significant unmet medical need that momelotinib’s triple-pathway inhibition mechanism may address.<\/p>\n\n\n\n

Regulatory Milestone Overview<\/h2>\n\n\n\n
Item<\/th>Detail<\/th><\/tr><\/thead>
Company<\/strong><\/td>GlaxoSmithKline plc (GSK) (NYSE: GSK)<\/td><\/tr>
Announcement Date<\/strong><\/td>12 June 2026<\/td><\/tr>
Drug Name<\/strong><\/td>Momelotinib (Ojjaara\/Omjjara)<\/td><\/tr>
Designation<\/strong><\/td>Orphan Drug Designation (ODD)<\/td><\/tr>
Regulatory Agencies<\/strong><\/td>US FDA and European Medicines Agency (EMA)<\/td><\/tr>
Indication<\/strong><\/td>VEXAS syndrome<\/td><\/tr>
Current Approvals<\/strong><\/td>US, EU, UK, Japan for myelofibrosis<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n

Disease Profile & Unmet Medical Need<\/h2>\n\n\n\n

VEXAS Syndrome Characteristics<\/h3>\n\n\n\n