Strong Revenue Performance
Total revenue for the third quarter was $197 million, including $124 million from the DMD franchise and $52 million from Emflaza. The company's total cash position exceeded $1 billion.
NDA Submissions and Approvals
PTC submitted two NDAs to the FDA, for sepiapterin and Translarna, both of which have been accepted for filing. Sepiapterin's NDA includes significant efficacy evidence and is under review in multiple regions.
Significant Pipeline Progress
Fast track designation was received for PTC518 for Huntington's disease. The company is on schedule to submit an NDA for vatiquinone, based on significant long-term benefits observed in trials.
Global Expansion and New Market Entries
Translarna received first-time orders from two new countries in the Middle East and North Africa region. The sepiapterin global launch is planned for 2025 with submissions under review in multiple countries.