Business
PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2024 Financial Results
– Full year 2024 revenue of $807 million, exceeding guidance – – All 2024 clinical and regulatory milestones were achieved on schedule, including four NDA
About this update from Ptc Therapeutics, Inc.
– Full year 2024 revenue of $807 million, exceeding guidance – – All 2024 clinical and regulatory milestones were achieved on schedule, including four NDA submissions, all of which were accepted for filing – – License and collaboration agreement with Novartis for PTC518 Huntington's disease program closed in January 2025 – – Cash of over $2.0 billion as of January 2025 – WARREN, N.J., Feb. 27, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter ending December 31, 2024. "Our strong fourth quarter rounds out a year of significant accomplishment across every part of our company," said Matthew B. Klein, M.D., Chief Executive Officer. "In 2024, our commercial team delivered another outstanding performance, we achieved all clinical and regulatory milestones on schedule and we solidified our balance sheet. We now have over $2 billion in cash to support our planned commercial and R&D activities in 2025 and beyond. With the many accomplishments of 2024 and our team's demonstrated ability to execute across every part of the business, we have built a strong foundation for continued success." Key Corporate Updates: Fourth quarter 2024 total revenue of $213 millionFourth quarter 2024 revenue for the DMD franchise of $144 million, including net product revenue for Translarna™ of $94 million and for Emflaza® of $50 millionSold Rare Disease PRV received with FDA approval of Kebilidi™ for $150 millionLicense and collaboration agreement signed with Novartis for PTC518 Huntington's disease program, closed in January 2025Key Clinical and Regulatory Milestones: PTC submitted four regulatory approval applications to FDA in 2024, all of which have been accepted for review:Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the treatment of AADC deficiency in the U.S., approved in November 2024Sepiapterin for children and adults with PKU, with a target regulatory action date of July 29, 2025Vatiquinone for children and adults with Friedreich's ataxia, granted priority review with a target regulatory action date of August 19, 2025Translarna for nmDMDPTC submitted several additional marketing authorization applications outside the U.S. for sepiapterin in 2024, with CHMP opinion on sepiapterin MAA expected in Q2 2025 and a regulatory decision in Japan expected in Q4 2025.Type C meeting wit...
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