Emcitate (tiratricol)
MCT8 deficiency (Allan-Herndon-Dudley syndrome) · Patients with monocarboxylate transporter 8 deficiency
EGTX · Egetis
Regulatory designations
Full timeline
4 milestonesFDA decision expected
FDA assigned a September 28, 2026 PDUFA target action date to the Emcitate NDA.
Egetis — NDA acceptance, Priority Review and PDUFA date↗FDA accepted the NDA with Priority Review
FDA accepted the filing and granted Priority Review; Egetis said no advisory committee meeting was then planned.
Egetis — NDA acceptance and Priority Review↗Egetis completed the rolling NDA submission
The completed application included clinical studies, real-world evidence and the U.S. expanded-access experience in MCT8 deficiency.
Egetis — Rolling NDA submission completed↗Egetis reached alignment with FDA on the NDA package
Following a pre-NDA meeting, Egetis said FDA agreed to a rolling submission based on the available clinical-data package.
Egetis — Pre-NDA meeting and rolling-review plan↗Related News
No related news available.