Acer repurposes drugs with available data (like a 40-year-old European antihypertensive) into rare disease treatments (like Edsivo for vascular Ehlers-Danlos syndrome), but approval remains elusive, with a CRL for ACER-001 just as Edsivo is poised to enter a new clinical trial, armed with a new breakthrough designation. The Pink Sheet delves into regulatory history and strategy with CEO Chris Schelling.

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

The company announced data from the Phase III SPRING study of Takhzyro in children aged 2-11; there is no approved long-term prophylactic therapy for children under 6.

The UK biotech looks to have reached the end of the road, despite some encouraging signs from its microbiome-based pipeline.