CAMBRIDGE, Mass. — A drug under investigation as a treatment for a rare genetic disorder that causes abnormally high triglyceride levels in the blood has received orphan-drug designation from the Food and Drug Administration.
Aegerion Pharmaceuticals announced that it received the designation for lomitapide as a potential treatment for familial chylomicronemia. The drug currently is in phase-3 trials as a treatment for homozygous familial hypercholesterolemia, and Aegerion said it expects to start a separate clinical trial to evaluate the drug for familial chylomicronemia in the second half of this year.
“The FDA’s grant of orphan-drug designation to lomitapide for the treatment of FC represents an important milestone in the overall development and commercialization plan for the compound, and it underscores the unmet need that exists,” Aegerion CEO Marc Beer said. “With this announcement, we are another step closer toward our goal of [treating] patients with these rare lipid disorders that currently have inadequate treatment options available.”
The FDA gives orphan-drug designation to treatments for diseases that affect fewer than 200,000 people in the United States. The designation includes tax incentives, possible exemption from the FDA user fee and a seven-year market exclusivity period upon approval, compared with the usual five years.