PODCAST | Regulation

A DMD decision & endpoint innovation: a BioCentury podcast

Plus: FDA’s single trial guidance, the Promising Pathway Act and Lexeo’s NASDAQ debut

November 7, 2023 1:19 AM UTC

The controversy around Sarepta Therapeutics Inc. (NASDAQ:SRPT) continues as the company’s executives were confident last week that the data from the Phase III EMBARK trial of Duchenne muscular dystrophy gene therapy Elevidys delandistrogene moxeparvovec were enough to warrant full approval despite missing the study’s primary endpoint. On the latest BioCentury This Week podcast, BioCentury’s editors analyze the pitfalls facing FDA as the agency considers whether to apply regulatory flexibility in its decision-making and the impact the decision could have for patients and future therapies that are submitted to the agency with less than clear-cut positive data.

The editors also discuss a package of articles published last week on innovations in evidence generation for new therapies, including the path forward for expanding use of digital endpoints and why pharmas are beginning to adopt tokenization and data linkage to more efficiently capture longitudinal data. The editors debate the impact that FDA’s guidance on approving drugs based on a single trial plus confirmatory evidence will have on rare disease drug development, and how that standard relates to proposed U.S. legislation that seeks to establish a provisional approval pathway. 

Finally, the editors assess the tepid reception for the latest biotech IPO, the debut by Lexeo Therapeutics Inc. (NASDAQ:LXEO), which came in the midst of last week’s mini-rally for biotech stocks.