Alirocumab in high-risk patients: Observations from the open-label expanded use program

Area of Science:

• Cardiology
• Pharmacology
• Genetics

Background:

• Severe hypercholesterolemia requires advanced lipid-lowering therapies.
• Standard therapies are often insufficient for high-risk patients.
• Alirocumab targets PCSK9 to reduce LDL-C.

Purpose of the Study:

• Evaluate alirocumab's safety and efficacy in high-risk patients.
• Assess early use of proprotein convertase subtilisin/kexin type 9 inhibitors.
• Provide real-world data before commercial availability.

Main Methods:

• Open-label alirocumab (150 mg every 2 weeks) for 24 weeks.
• Enrolled patients with heterozygous familial hypercholesterolemia (HeFH) and/or coronary heart disease (CHD).
• Included patients with baseline LDL-C ≥160 mg/dL on maximally tolerated lipid-lowering therapy, including statins or those with statin intolerance.

Main Results:

• 100 patients enrolled; 64% had CHD, 61% had HeFH.
• Alirocumab reduced LDL-C by 55% (from 221 mg/dL to 102 mg/dL).
• 61% experienced treatment-emergent adverse events; 3% led to discontinuation; no deaths.

Conclusions:

• Alirocumab demonstrated significant LDL-C reduction in high-risk patients.
• Safety and efficacy align with findings from placebo-controlled ODYSSEY trials.
• Supports alirocumab's role in managing severe hypercholesterolemia.