FDA recommends pre-BLA meeting for Rezolute's congenital HI drug despite missed trial endpoints
The agency's recommendation follows its review of additional data from the Phase 3 sunRIZE study of ersodetug, which did not meet its primary or key secondary endpoints.
Rezolute, Inc. said on October 5, 2026 that the FDA recommended a pre-BLA meeting with the company as a precursor to a biologics license application for ersodetug in congenital hyperinsulinism, after reviewing detailed data from the Phase 3 sunRIZE study.
The sunRIZE trial, a Phase 3 multicenter, double-blind, randomized, placebo-controlled study of ersodetug in congenital HI, showed glycemic improvements but failed to meet its primary endpoint, as the drop in hypoglycemia events measured by finger-stick self-monitored blood glucose was not statistically significant versus placebo.1 The key secondary endpoint, time in hypoglycemia measured by continuous glucose monitoring, also was not met at the Week 24 end-of-treatment window, though the company reported consistent improvements in this and other CGM-based measures across both treatment arms.1
At a March 2026 Type B meeting, the FDA acknowledged behavioral factors complicating trials in this population, including limits of self-monitored blood glucose measures, and asked Rezolute to submit continuous glucose monitoring data for independent review.1 Rezolute submitted source and analysis datasets along with results from numerous pre-specified, post-hoc, and sensitivity analyses in June 2026.1
Despite the trial missing its primary and key secondary endpoints, the FDA encouraged the company to request a Type B pre-BLA meeting, while stating it had not opined on approvability and that any such determination would come only during a formal BLA review.1
Rezolute said it expects topline results this quarter from its Phase 3 upLIFT study in tumor HI, and will decide on its overall BLA strategy for both indications after those results are announced.1
The company also noted that an open-label extension of sunRIZE is ongoing, with treatment durations ranging from about nine months to over two years, along with sustained glycemic control and reduced use of background standard-of-care therapies.1
Written by readthrough’s AI from the linked primary sources and fact-checked against them automatically before publishing. Not investment advice.