Vatiquinone

Larimar Therapeutics is progressing with clinical trials of nomlabofusp, targeting adolescent patients with Friedreich's ataxia, with potential data expected in mid-2025.

PTC Therapeutics anticipates a CHMP opinion on sepiapterin's MAA in Q2 2025, marking a crucial step toward potential European approval for PKU treatment.

The FDA has accepted PTC Therapeutics' NDA for vatiquinone, a potential treatment for Friedreich's ataxia (FA), granting it priority review.

PTC Therapeutics' Phase 2 CardinALS trial of utreloxastat in amyotrophic lateral sclerosis (ALS) failed to meet its primary endpoint of slowing disease progression.

PTC Therapeutics has submitted three approval applications to the FDA, including sepiapterin for PKU, Translarna for nmDMD, and a BLA for AADC deficiency gene therapy.