Possible Treatment for Homozygous Familial Hypercholesterolemia Earns Orphan Drug Designation

According to a story from bloomberg.com, the drug development company Arrowhead Pharmaceuticals, Inc. recently announced that its experimental product candidate AR0-ANG3 has earned Orphan Drug designation from the US Food and Drug Administration (FDA). The drug was given this designation as a treatment for homozygous familial hypercholesterolemia, a genetic condition that causes increased cholesterol levels.

About Familial Hypercholesterolemia

Familial hypercholesterolemia is a condition of elevated, abnormally high levels of LDL cholesterol that is caused by a hereditary genetic mutation. The mutation that causes the condition affects the LDLR gene which normally codes for a protein that removes LDL from the bloodstream. The primary symptoms are elevated LDL cholesterol, deposits of cholesterol in certain areas of the body (eyelids, iris, and tendons of the arms and legs), and early onset cardiovascular issues, such as coronary artery disease, strokes, heart attack, and atherosclerosis. While once considered rare, it is likely that many people with the condition live undiagnosed. Treatment may include statins or other medications, liver transplant, or other surgical operations. The true prevalence of familial hypercholesterolemia remains unknown. To learn more about familial hypercholesterolemia, click here.

Homozygous familial hypercholesterolemia is considered to be the more difficult form of the condition to treat. It often requires high doses of statins and other drugs but for some patients these are only modestly effective. There are only a very limited number of drugs that are approved for this form of the disease.

About Orphan Drug Designation

Orphan Drug designation is typically reserved for therapies that are being developed to treat diseases that are considered rare, which is defined as any illness that affects less than 200,000 people in the US. To qualify, the drug must fulfill a currently unmet medical need or offer substantial advantages in safety or effectiveness in comparison to current medications. The designation confers several benefits to the recipient company, such as the waiving of certain fees, tax breaks, and a seven year period of market exclusivity if the drug is approved for public use.

AR0-ANG3 is a RNAi interference based therapeutic that is currently being developed for homozygous familial hypercholesterolemia and other metabolic illnesses. It is currently being tested in healthy subjects in a phase 1 clinical study.


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