Shelby Hill's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q2 2025
Question
On behalf of Luca Eci from RBC Capital Markets, Shelby Hill asked for more color on the Sanfilippo CRL, referencing prior comments about shipping validation and a stringent inspector, and questioned if the FDA was 'missing the forest for the trees.'
Answer
Emil Kakkis, Founder, President & CEO, responded that it is the FDA's job to scrutinize details ('check every tree') and that CMC rigor is essential. He emphasized that the company's focus is on methodically addressing all the issues raised in the CRL, which they believe are entirely manageable, rather than dwelling on the inspection process.