Data from part B of the Swedish firm’s pivotal trial of Tarpeyo in the rare kidney disorder showed that the treatment effects lasted two years across the entire population, enabling the move from conditional to full approvals in several markets.

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker

Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.

Regulatory reliance is playing an increasingly important role in gaining marketing authorization in the UK following Brexit. Meanwhile, the country’s unique Innovative Licensing and Access Pathway is attracting growing levels of interest, not just from companies but from regulators and health technology appraisal bodies across the world.