HoFH is a rare genetic disorder that results in extremely high cholesterol. Patients with this disease display signs of high cholesterol early in life and may experience heart attacks from early childhood.
Isis claims that treatment for HoFH is currently inadequate and many patients undergo an expensive and time consuming procedure called apheresis, a process similar to kidney dialysis to remove the “bad” cholesterol from the blood.
The orphan drug status, which provides funding for drug development for diseases affecting fewer than 200,000 people in the US, entitles Isis to seven years of market exclusivity for ISIS 301012
Additional incentives include tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.
“This orphan drug designation underscores the need for improved therapies for familial hypercholesterolemia (FH). Isis is committed to rapidly developing ISIS 301012 for FH, as well as more routine forms of high cholesterol,” said Dr Mark Wedel, senior vice president of development and chief medical officer at Isis.