Pasadena Pharmaceutical Company’s New Drug Helps People with Rare Fat Disorder

Pasadena-based Arrowhead Pharmaceuticals announced Sunday that its experimental drug plozasiran significantly reduced triglycerides and the risk of acute pancreatitis in patients who have a rare genetic disorder which currently has no Food and Drug Administration-approved treatment in the U.S.

Arrowhead reportedly tested plozasiran on 75 people with this rare condition, which is called familial chylomicronemia syndrome, or FCS for short. People with FCS have very high levels of fat in their blood, which can cause painful attacks in their pancreas.

But the new drug seems to work well, Arrowhead reported. It lowered the amount of fat in people’s blood by about 80%. It also made the painful pancreas attacks much less likely to happen, the company said in  statement.

Gerald F. Watts, Ph.D., professor of cardio-metabolic medicine at the University of Western Australia, called the results “encouraging” and said they “offer hope to people living with FCS and their physicians who are in desperate need of new safe and effective treatment options.”

The most common side effects included abdominal pain, COVID-19, nasopharyngitis, headache, nausea, back pain, upper respiratory tract infection, and diarrhea.

Based on these findings, Arrowhead plans to file a New Drug Application with the Food and Drug Administration by the end of 2024 and seek regulatory approval with additional global authorities thereafter.

The results were presented at the European Society of Cardiology Congress 2024 and simultaneously published in The New England Journal of Medicine.

Arrowhead planned to host a virtual analyst and investor event in the early morning Tuesday, September 3 to discuss the results.