The Medicines and Healthcare products Regulatory Agency (MHRA) has approved?olezarsen (tryngolza) to help treat adults with Familial Chylomicronemia Syndrome (FCS).

FCS is an inherited disease that gives rise to abnormally high levels of fats called triglycerides in the blood. This can lead to inflammation of the pancreas, causing severe pain, lasting damage to the pancreas, and can be life threatening.

Olezarsen is administered as an injection under the skin, usually into the stomach area, the front of the thighs, or the back of the upper arms.

In a main study involving 66 adults with FCS, olezarsen was shown to significantly reduce triglyceride levels in the blood. All patients followed a controlled diet and received either Tryngolza or a placebo. 

After six months, patients treated with Tryngolza saw an average reduction in triglycerides of 32 percent, compared with an average increase of 12 percent in those given placebo. These benefits were maintained and further improved after one year, with fewer cases of acute pancreatitis reported in patients taking tryngolza.

Speaking in this regard, Julian Beach, Interim Executive Director—Healthcare Quality and Access, MHRA, said, “This approval provides a new treatment option for adults living with Familial Chylomicronemia Syndrome, an inherited condition that can lead to inflammation of the pancreas, causing severe pain and potentially life-threatening complications.

“As with all medicines, we will continue to closely monitor the safety and effectiveness of olezarsen as it is used more widely.”