PTC Therapeutics' Waylivra Scores Approval In Brazil For Genetic Disorder

The Brazilian Health Regulatory Agency has approved PTC Therapeutics Inc's PTCT Waylivra (volanesorsen) as the first treatment for familial chylomicronemia syndrome (FCS).
The approval is based on the Phase 3 APPROACH study results and the APPROACH Open-Label Extension study and is supported by results from the Phase 3 COMPASS study.
Results from the Phase 3 APPROACH trial showed that the treatment reduced triglycerides by 94% compared to placebo. All patients in the trial maintained a low-fat diet.
Waylivra is a self-administered, subcutaneous injection that comes in a single-use, prefilled syringe.
Waylivra (volanesorsen), a product of Ionis Pharmaceuticals Inc's IONS antisense technology, is conditionally approved in the E.U. for FCS.
FCS is a genetic disease caused by impaired function of the enzyme lipoprotein lipase and characterized by severe hypertriglyceridemia and risk of unpredictable and potentially fatal acute pancreatitis.
Related content: Benzinga's Full FDA Calendar.
Price Action: PTCT shares closed 3.48% higher at $39.53 on Monday.