PTC Therapeutics has just announced that their therapy Waylivra (volanesorsen), a treatment for familial chylomicronemia syndrome (FCS), has been given Category 1 classification from the Drug Market Regulation Chamber in Brazil. It is now the only therapy for FCS in the country and this new classification provides pricing to match international markets.
PTC Therapeutics has also submitted their application of Waylivra to the Brazilian Health Regulatory Agency as a therapy for familial partial lipodystrophy (FPL). If approved, the drug will become the very first approved therapy for FPL in the country. It would also be the very first approval of this therapy for the condition internationally. PTC expects a decision on their application by the 2nd half of this year.
Waylivra, or volanesorsen, is an antisense technology. It has already been given conditional marketing approval and Orphan Drug designation in the EU for FCS.
Familial Chylomicronemia Syndrome
This is a rare condition which is the result of a malfunctioning lipoprotein lipase (LPL) which means it cannot properly break down chylomicrons. FCS patients face severe hypertriglyceridemia, and a high risk of acute pancreatitis, which can be fatal. Other complications include fatigue, abdominal pain, and impaired cognition.
Due to these symptoms, quality of life is effected and psychosocial variables are impacted for many patients. Many FCS patients face depression, social withdrawal, brain fog, and anxiety.
Further, severe and necessary lifestyle changes impact day to day life. Patients must eat a very low-fat diet to manage their disease due to a lack of pharmaceutical treatments. That said, even this treatment is not enough and does not eliminate pancreatitis risk.
Familial Partial Lipodystrophy
FPL is a condition which leads to a progressive decrease of adipose tissue (fat) from selective parts of the patient’s body. Patients face fat deposition in the muscle and the liver. Additionally, insulin resistance, fatty liver disease, diabetes, and dyslipidemia can occur.
Typical presentation is a reduction of subcutaneous fat in the extremities and an excess of fat in the neck, intraabdominal area, and face.
This condition is highly burdensome, and physically difficult to manage. Current therapies do not properly manage the metabolic complications of this disease.
You can read more about these conditions and the potential new treatment here.