External Comparator Alignment RiskFurther alignment with regulators on how trial results will be compared to the external FACOMS database remains necessary and creates uncertainty around approval comparisons.
Regulatory Timing And Filing RiskUnresolved issues over trial comparisons and endpoints could lead to delays in filing acceptance or extend the overall review timeline, affecting near-term approval prospects.
Surrogate Endpoint Acceptance RiskRegulatory acceptance of skin frataxin levels as a surrogate marker for symptoms is not guaranteed and may require additional evidence or design changes to satisfy reviewers.