According to a story from globenewswire.com, Akcea Therapeutics and Ionis Pharmaceuticals recently announced the release of top line results from a clinical study. This trial tested the drug WAYLIVRA as a treatment for familial partial lipodystrophy (FPL), a rare disease. The study results suggest that this drug could be a useful treatment for the disease as the drug was able to achieve the primary study endpoint. The results are good news for patients dealing with this illness, which is lacking in effective treatment options.
About Familial Partial Lipodystrophy (FPL)
Familial partial lipodystrophy, which is also known as Köbberling-Dunnigan syndrome, is a rare disease most characterized by abnormal loss of body fat from various areas of the body. There are several different subtypes of this disease which are distinguished from each other primarily by which genetic mutation is responsible. There are five separate genes that, when mutated, can cause the disorder. Symptoms often appear at the beginning of puberty. These symptoms include fat loss in the arms, legs, and abdomen; abnormal buildup of fat in other areas of the body, such as the neck, face, and shoulders. Other symptoms may include elevated blood triglycerides, diabetes, glucose intolerance, pancreatitis, heart problems, and cirrhosis. Prognosis and disease severity can vary widely. Treatment is primarily based on the symptoms present. A diet high in carbs and low in fat is beneficial as is regular exercise. To learn more about familial partial lipodystrophy, click here.
Trial Results
The trial included a total of 40 patients aged 18 or older with familial partial lipodystrophy. These patients also presented with fatty liver and elevated triglycerides. WAYLIVRA was delivered via an injection under the skin. The study period lasted for one year and during this time patients saw a mean reduction in triglycerides of 88 percent. In addition, liver fat was reduced by a mean of 51.9 percent. There were no serious adverse events recorded during the study.
These encouraging results indicate that WAYLIVRA, which is currently approved in the EU to treat the rare disease familial chylomicronemia syndrome (FCS), has significant capability in treating familial partial lipodystrophy as well. Hopefully, WAYLIVRA will ultimately be approved for this indication.
