Rezolute reports that the US Food and Drug Administration has suggested a pre-BLA meeting for ersodetug, following a mixed Phase 3 trial in congenital hyperinsulinism, as the company prepares for potential approval in a high-need rare disease area.
Rezolute said the US Food and Drug Administration has recommended a pre-BLA meeting as the next step towards a possible biologics licence application for ersodetug in congenital hyperinsulinism, after reviewing additional data from the drug’s Phase 3 sunRIZE study. The company said the agency did not comment on whether the treatment would ultimately be approvable, and that any decision would come only after a formal review of a complete application.
The announcement follows a mixed read-out from sunRIZE, in which Rezolute said the trial showed better blood-sugar control but missed its main target because reductions in hypoglycaemic episodes measured by finger-stick self-monitoring were not statistically significant against placebo. The company also said the key secondary endpoint, based on continuous glucose monitoring, was not met in the Week 24 assessment window, although it argued that other monitoring measures showed clinically meaningful improvements.
The FDA’s response appears to fit with its broader approach to rare disease drug development, where regulators often seek detailed discussions with sponsors over what evidence can support approval in small patient populations with high unmet need. The agency’s rare disease guidance and its biologics centre both emphasise early scientific and regulatory input, while a separate evidence-principles programme has been designed to give sponsors clearer direction on the kinds of data that may be considered in rare disorders.
Rezolute also said it expects topline results from its Phase 3 upLIFT study in tumour-related hyperinsulinism later this quarter, and that it may decide on a broader filing strategy after those findings. The company said the open-label extension of sunRIZE is continuing, with patients remaining on treatment for periods ranging from about nine months to more than two years, and with ongoing glycaemic control and lower use of background therapy. Julie Raskin, chief executive of Congenital Hyperinsulinism International, said families still face severe and unpredictable hypoglycaemia and welcomed progress towards possible new options.
Disclaimer: This content is for informational purposes only and is not intended to be a substitute for professional medical judgment, advice, diagnosis, or treatment.
