PTC Therapeutics, Inc. (PTCT) Launches SUFIANCE for PKU

Company Overview - PTC Therapeutics, Inc. (NASDAQ:PTCT) is focusing on rare disease innovation with the recent launch of SUFIANCE (sepiapterin) for phenylketonuria (PKU) [2] - The drug has been approved by both the FDA and EU, with a rollout in Germany in July 2025 and an imminent launch in the U.S. [2] Financial Performance - SUFIANCE is projected to generate over $1 billion annually in the U.S., serving as a central driver of PTCT's growth [2] - The company has $1.9 billion in cash reserves, which supports a path toward cash-flow breakeven [4] Pipeline Challenges - PTC Therapeutics is facing challenges with its pipeline, as the FDA issued a Complete Response Letter for vatiquinone, its candidate for Friedreich's ataxia, requesting additional clinical data [3] - The company is working closely with regulators to address these concerns while maintaining progress in its broader rare disease studies [3] Strategic Initiatives - To accelerate the adoption of SUFIANCE, the company is leveraging early access programs and strategic partnerships to expand its global reach [2] - The company continues to advance its pipeline, including promising gene therapy programs such as PTC518 for Duchenne muscular dystrophy (DMD) [4]