Core Viewpoint - The U.S. FDA has approved PTC Therapeutics' PTCT SEPHIENCE (sepiapterin) for treating phenylketonuria (PKU), a rare genetic disorder, which is expected to significantly impact the company's future revenue and position in the market [1][4][7]. Group 1: Product Approval and Market Potential - The approval includes broad labeling for hyperphenylalaninemia (HPA) in patients aged one month and older with sepiapterin-responsive PKU [2]. - The FDA's decision is based on significant efficacy and safety data from the Phase 3 APHENITY trial and its long-term extension study [4]. - PTC Therapeutics anticipates a potential revenue opportunity exceeding $1 billion for Sephience [6]. Group 2: Commercial Strategy - The initial commercial launch strategy will target 1,200 prescribers across 104 centers, covering over 80% of PKU claims data in the U.S. [4]. - The wholesale acquisition cost (WAC) for Sephience is approximately $40,000 per month or $480,000 per year [5]. - The company will prioritize patients who have not responded well to existing oral therapies, estimated to include 40% of the PKU population [5][6]. Group 3: Analyst Insights and Stock Performance - Analysts view the approval as a significant achievement for PTC, with expectations that Sephience could become the new standard of care for PKU patients [7]. - William Blair projects peak global sales for Sephience to reach $2.2 billion by 2031 and total revenue of $1.71 billion in 2025 [7]. - PTC stock has seen a price increase of 12.44%, reaching $49.86 at the time of publication [9].