PTC Therapeutics provided a corporate update on January 13, 2025, announcing unaudited total revenue for 2024 of approximately $814 million, which exceeded its previously issued guidance. The company highlighted its strong performance across all areas, including meeting every planned clinical and regulatory milestone on schedule in 2024.
In 2024, PTC submitted four approval applications to the FDA, all of which were accepted for review, demonstrating significant pipeline advancement. The global license and collaboration agreement with Novartis for the PTC518 Huntington's disease program officially closed in January 2025, further strengthening the company's financial position.
PTC Therapeutics reported a strong cash balance at the start of 2025 and provided financial guidance for the year, projecting non-GAAP research and development and selling, general, and administrative expenses between $730 million and $760 million. The company is preparing for global launches of sepiapterin for PKU, which represents a potential $1 billion market opportunity.
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