According to a story from Bioportfolio, trials testing the treatment inclisiran are reaching their enrollment goals ahead of schedule. Inclisiran is currently being tested to reduce LDL-cholesterol in a variety of conditions, such as in patients with atherosclerotic cardiovascular disease and familial hypercholesterolemia. The treatment has already shown promise in earlier Phase II trials. News trials will test the drug’s tolerability and safety.
Familial hypercholesterolemia (FH) is a genetic disorder in which the patient has abnormally high levels of low-density lipoprotein (LDL) cholesterol in the body. They also tend to suffer from cardiovascular disease earlier in their lives than most people. While the high cholesterol characteristic of familial hypercholesterolemia does not produce symptoms on its own, people with the disorder may have unusual deposits of cholesterol rich fat in the different areas of the body. The area above the eyelids is a common location for these deposits. Most people with FH are undiagnosed, but their likelihood of cardiovascular problems is much higher and they can develop much earlier in life. People with FH often have to take high doses of statins as treatment. To learn more about FH, click here.
Trials testing the effectiveness of inclirisan on FH patients are expected to complete enrollment by the first half of the year. The trials are being conducted by The Medicines Company, and inclisiran has already been given orphan drug status by the Food and Drug Administration (FDA). Orphan drug status will allow for the pace of development for the treatment to increase and gives the company exclusive rights to develop to the treatment as well as a seven year period of market exclusivity. Orphan drug status is only given to treatments that promise a major breakthrough in treatment effectiveness or that serve a medical need that is not being met by current publicly available medications.
