Phase III ODYSSEY ESCAPE trial evaluating Praluent (alirocumab) in heterozygous familial hypercholesterolemia meets primary endpoint- Sanofi/Regeneron

Sanofi and Regeneron Pharmaceuticals announced positive results from the Phase III ODYSSEY ESCAPE trial evaluating Praluent (alirocumab) Injection in patients with an inherited form of high cholesterol known as heterozygous familial hypercholesterolemia (HeFH), whose cholesterol levels required chronic, weekly or bi-weekly apheresis therapy. The trial met its primary endpoint, demonstrating that Praluent plus existing treatment regimen can significantly reduce the frequency of apheresis therapy by 75 percent, compared to placebo. Sixty-three percent of patients treated with Praluent no longer required apheresis, compared to zero percent of placebo patients.

Apheresis is a procedure where bad (LDL) cholesterol is removed from the blood, in a process similar to kidney dialysis. The most common adverse events in the trial were fatigue (15 percent Praluent; 10 percent placebo) and nasopharyngitis (10 percent Praluent; 10 percent placebo). Detailed data will be presented at future medical congresses.

Comment: Despite statins, a subset of patients with heterozygous familial hypercholesterolemia are unable to sufficiently reduce their LDL cholesterol, and require regular apheresis treatment. This therapy is invasive and burdensome to patients, given that it can take more than three hours. Treatment may also be inconvenient and cost up to $100,000 for each patient per year in the U.S.