Clinical TrialsThe Phase II CardinALS clinical trial of utreloxastat in ALS failed to meet primary and secondary efficacy endpoints, resulting in no further development plans.
Financial PerformanceThe expected total revenue for 2025 is anticipated to decrease compared to the previous year, with guidance between $600 million and $800 million.
Regulatory ChallengesTranslarna's New Drug Application was accepted for review, but the FDA's lack of timeline and uncertain approval due to regulatory history raises concerns.