Strong Start to 2025 with $190 Million Revenue
PTC Therapeutics achieved $190 million of revenue in the first quarter of 2025, with strong contributions from the DMD franchise.
SUFIANCE's Anticipated Global Launch
Positive CHMP opinion on SUFIANCE marketing authorization with an expected broad label for all PKU patients. Preparations for European and U.S. launches are underway, aiming for over $1 billion in revenue.
PTC518 Huntington’s Disease Program Success
The PIVOT HT Phase 2 study met its primary endpoints of blood HTT lowering and safety, with continued trends of dose-dependent favorable clinical effects after 24 months of treatment.
Strong Cash Position
PTC closed the first quarter with over $2 billion in cash, allowing support for all planned commercial and R&D activities without needing additional capital.
Vatiquinone NDA Progress
FDA review of vatiquinone for Friedreich’s ataxia is progressing well, with no plans for an AdCom meeting, indicating confidence in the approval process.
DMD Franchise Revenue Defense
Delivered strong first-quarter revenue of $134 million for the global DMD franchise, demonstrating effective defense strategies despite challenges.