Financial Performance - Unaudited total revenue for 2024 was approximately 814million,exceedingguidance[1]−Unauditednetproductrevenuefor2024wasapproximately601 million, driven by the DMD franchise with 340millionfromTranslarnaand207 million from Emflaza[3] - Cash balance as of December 31, 2024, was approximately 1.1billion,withanadditional1.0 billion from the PTC518 collaboration agreement with Novartis[3] - PTC anticipates approximately 211millioninroyaltyrevenuefromEvrysdiforthefullyear2024[8]FutureProjections−PTCexpectstotalrevenuesfor2025tobebetween600 million and 800 million, including in-line products and potential new product launches[8] - Anticipated GAAP R&D and SG&A expenses for 2025 are projected to be between 805 million and 835million[8]RegulatoryApprovals−FourregulatoryapprovalapplicationsweresubmittedtotheFDAin2024,includingsepiapterinforPKUwithanFDAactiondateofJuly29,2025[3]−CHMPopiniononsepiapterinMAAisexpectedinQ22025[8]CollaborationsandMilestones−PTCsignedagloballicenseandcollaborationagreementwithNovartisforPTC518,withpotentialmilestonepaymentsupto1.9 billion[3] - Results from the PIVOT-HD Phase 2 study of PTC518 are expected in Q2 2025[8]