Benjamin P. Saylor

The July 1, 2026 decision makes exagamglogene autotemcel the first gene therapy approved for SCD in children younger than 12 years.

Among children treated with navepegritide throughout the double-blind period, mean TFA decreased from 9.1° at baseline to 7.7° at Week 52 and 6.9° at Week 104, a mean absolute change of −2.2° over 2 years.

In a parallel regulatory action, the European Medicines Agency has also validated a marketing application for Tivicay covering the neonatal indication.

Cadrenal has announced that it plans to seek FDA Rare Pediatric Disease Designation for tecarfarin in children with Kawasaki disease and coronary aneurysms.

Baloxavir marboxil is a first-in-class cap-dependent endonuclease inhibitor that functions as a prodrug.

The approval is based on data from the phase 3 STRIDE-13 trial.

New Brain Communications data show doxecitine and doxribtimine reduce mortality risk by up to 94% in early-onset thymidine kinase 2 deficiency.