US Biotech Surpasses Guidance for New Rare Disease Treatment Despite DMD Drug Setback

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on the 19th that its 2025 product and royalty revenues totaled $831 million, surpassing the guidance set earlier this year, even as it voluntarily withdrew its U.S. NDA resubmission for Translarna, a candidate treatment for Duchenne muscular dystrophy (DMD). The company said it has secured approximately $1.95 billion in cash and marketable securities by combining projected 2025 revenue of $111 million from its new rare disease therapy Sepience, approvals in Japan and Brazil, and $240 million from the sale of its remaining Evrysdi royalty interest. For 2026, PTC forecasts product revenues of $700 million to $800 million and R&D plus SG&A expenses of $775 million to $815 million. Earlier, on the 12th, the U.S. Food and Drug Administration informed the company that Translarna’s clinical data was unlikely to meet efficacy standards, prompting PTC to halt its U.S. relaunch efforts and creating additional global regulatory and commercialization uncertainties for the drug. (Source: pharmexec.com)

Following the Translarna withdrawal, PTC’s share price underwent a short-term correction. However, recent overseas market analyses suggest that while the decision is a setback for its DMD portfolio, it could refocus attention on Sepience and other rare-disease assets. Data aggregated by investment-information firms show that Wall Street analysts continue to maintain a majority “Buy” rating on PTC, with a 12-month average target price implying double-digit upside from current levels. (Source: simplywall.st)

PTC is a U.S. biotech company developing and commercializing therapies for rare genetic disorders such as DMD, phenylketonuria (PKU), Huntington’s disease, and Friedreich’s ataxia. Its core pipeline includes the DMD treatments Translarna and Emflaza, as well as the PKU therapy Sepience. (Source: prnewswire.com) The rare-disease drug market—characterized by small patient populations but high pricing and stringent regulatory requirements—is highly sensitive to FDA review outcomes and global approval and reimbursement decisions, with the success or failure of a single asset having a major impact on corporate performance and stock valuation. (Source: pharmaceutical-technology.com)