Ultragenyx Pharmaceutical Inc.

Plus: BG Rhee joining Flagship, a Jeito promotion, a new CEO for Ashvattha and more

Plus: Ultragenyx hires Stoke’s Olsen as new CBO, CFO transition at CSL and updates from MannKind, enGene, Altimmune

Also in BioCentury’s clinical report, heparan sulfate gets more clinical validation in MPS, but will the biomarker ever be a surrogate endpoint?

Plus: Anne White retiring as president of Lilly Neuroscience and updates from Parent Project Muscular Dystrophy, Opthea, Cantargia, Scilex and more

When even strong biomarker science isn’t accepted and review timelines slip, children lose time — and investors lose confidence

The agency must act with urgency and not be trapped by standards that can only be met in large indications

Despite some recent delays, median response time is one day before PDUFA

Company is latest to be surprised by agency’s concerns over issues not previously raised 

Plus: Astrazeneca and Takeda preparing submissions after Phase III readouts, in this week’s Clinical Report

But biomarker heparan sulfate likely to serve supportive role, not the basis of accelerated approval, according to CEO