PTC Therapeutics, Inc. announced that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil. This is the first approval globally for Waylivra for the FPL indication. Waylivra is also approved in Brazil for the treatment of Familial Chylomicronemia Syndrome (FCS).
“Waylivra’s approval as the first treatment for FPL is an important milestone for FPL patients in Brazil,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “This approval is another example of PTC leveraging its strong capabilities in Latin America to bring first of its kind transformational therapies to patients with high unmet needs.”
FPL is a rare genetic metabolic disease characterized by selective, progressive loss of body fat (adipose tissue) from various areas of the body leading to ectopic fat deposition in liver and muscle and development of insulin resistance, diabetes, dyslipidemia, and fatty liver disease1. FPL is a highly burdensome disease, leading to significant metabolic complications that are not managed by current therapies, and it is a distressing condition for the patient.
ANVISA’s approval for Waylivra for FPL was based on results from the Phase 2/3 BROADEN study in patients with familial partial lipodystrophy. The study met its primary endpoint demonstrating a statistically significant reduction in triglyceride levels in patients with FPL treated with Waylivra compared to placebo-treated patients.
In the study, there was a mean reduction from baseline of 88 percent in triglyceride levels in patients treated with Waylivra compared to 22 percent reduction in placebo-treated patients at three months. Clinically meaningful and statistically significant reductions in triglyceride levels were sustained over 12 months of treatment in FPL patients. In addition to achieving the primary endpoint, the study achieved an important secondary endpoint of a statistically significant reduction in liver fat.