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Obicetrapib in patients with heterozygous familial hypercholesterolemia: the BROOKLYN randomized clinical trial

Stephen J Nicholls1, Adam J Nelson2, Marc Ditmarsch3

  • 1Victorian Heart Institute, Monash University, Clayton, Victoria, Australia. stephen.nicholls@monash.edu.

Nature Medicine
|February 27, 2026
PubMed

Insights

Obicetrapib effectively lowers LDL cholesterol in patients with familial hypercholesterolemia. This novel therapy offers significant lipid reduction when maximally tolerated treatments are insufficient.

Area of Science:

  • Cardiovascular Medicine
  • Pharmacology
  • Genetics

Background:

  • Familial hypercholesterolemia (FH) is a genetic disorder characterized by high LDL cholesterol.
  • Many FH patients do not reach target LDL cholesterol levels with current therapies.
  • A significant unmet need exists for effective treatments to manage FH.

Purpose of the Study:

  • To evaluate the safety and efficacy of obicetrapib in patients with heterozygous FH.
  • To assess the impact of obicetrapib on LDL cholesterol and other lipid parameters.
  • To determine if obicetrapib provides additional lipid lowering beyond maximally tolerated therapy.

Main Methods:

  • A randomized, placebo-controlled trial was conducted.
  • 354 patients with heterozygous FH and elevated LDL cholesterol were enrolled.
  • Participants received either 10 mg obicetrapib or placebo daily for 365 days, alongside maximally tolerated lipid-lowering therapy.

Main Results:

  • Obicetrapib demonstrated a significant placebo-adjusted reduction in LDL cholesterol of -36.3% at 84 days (P < 0.0001).
  • Secondary endpoints showed substantial reductions in apolipoprotein B (-24.4%), non-HDL cholesterol (-34.5%), and lipoprotein(a) (-45.9%).
  • A notable placebo-adjusted increase in HDL cholesterol of +138.7% was observed with obicetrapib.

Conclusions:

  • Obicetrapib is a safe and effective therapy for additional lipid lowering in patients with heterozygous FH.
  • The drug significantly improves multiple atherogenic lipid parameters.
  • Obicetrapib represents a promising new treatment option for managing FH.

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