NewAmsterdam and Menarini win EC approval for obicetrapib

The European Commission has approved obicetrapib as both a monotherapy and fixed-dose combination, marking the CETP inhibitor's first regulatory clearance worldwide.

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NewAmsterdam Pharma and Menarini Group have secured European Commission marketing authorisation for two obicetrapib-based products: Ubeslo (obicetrapib 10 mg monotherapy) and Evlarco (obicetrapib 10 mg plus ezetimibe 10 mg fixed-dose combination). Both products are indicated for adults with primary hypercholesterolaemia, covering heterozygous familial and non-familial forms, as well as mixed dyslipidaemia. The approvals, which follow a positive opinion from the EMA's Committee for Medicinal Products for Human Use, represent the first regulatory clearance of obicetrapib anywhere in the world.

Obicetrapib is an oral, once-daily, low-dose inhibitor of cholesteryl ester transfer protein (CETP), a mechanism that raises HDL while lowering LDL-C. The approval package draws on data from three Phase 3 trials: BROADWAY, BROOKLYN, and TANDEM. Monotherapy reduced LDL-C by up to 40% versus placebo; the fixed-dose combination with ezetimibe produced reductions of approximately 50% versus placebo. In both cases, tolerability was reported as comparable to placebo across the trial programme.

The commercial structure

Under the existing licensing agreement, Menarini holds exclusive commercialisation rights for obicetrapib across Europe. NewAmsterdam is entitled to tiered royalties ranging from low double-digits to mid-twenties on European net sales, together with up to an additional 833 million euros contingent on clinical, regulatory, and commercial milestones. Menarini, headquartered in Florence and present in 140 countries, brings an established cardiovascular sales infrastructure to the launch, which should accelerate uptake relative to what NewAmsterdam could achieve independently.

Michael Davidson, chief executive of NewAmsterdam, described the approval as validating "years of work focused on delivering a potential new treatment option for patients who continue to struggle to achieve recommended LDL-C levels despite previously available therapies." Elcin Barker Ergun, chief executive of Menarini, said the group would leverage its "well-established commercial capabilities and deep cardiovascular expertise" to reach eligible patients across the region.

Market context and competitive landscape

The approval arrives into a lipid-lowering market that is already well-populated but where unmet need remains substantial. Statins are the backbone of LDL management, yet a significant proportion of high-risk patients do not reach guideline-recommended targets. Ezetimibe provides modest additive benefit, and the PCSK9 inhibitor class, represented by evolocumab (Repatha, Amgen) and alirocumab (Praluent, Sanofi/Regeneron), offers injectable options with robust LDL-C reduction but adoption has been constrained by cost and administration route. Inclisiran (Leqvio, Novartis), an RNA interference therapy delivered by twice-yearly injection, has expanded the non-statin toolkit in Europe since its 2020 approval.

Obicetrapib's positioning as an oral, once-daily agent could appeal to patients and prescribers seeking an alternative to injectables, particularly in the familial hypercholesterolaemia population and in statin-intolerant individuals. Earlier CETP inhibitors from other developers, including torcetrapib and dalcetrapib, failed on cardiovascular outcomes or were discontinued. Obicetrapib's tolerability data, which so far mirror placebo, address some of the class's historical safety concerns, though the pivotal question of whether the drug reduces major adverse cardiovascular events will not be answered until the PREVAIL outcomes trial reads out. PREVAIL enrolled over 9,500 patients, completing enrolment in April 2024, and its results will be central to any broader adoption of obicetrapib by payers and clinical guidelines bodies. Additional Phase 3 trials, REMBRANDT and RUBENS, are also ongoing.

NewAmsterdam has indicated it intends to pursue regulatory approval in the United States and other markets, making the European clearance the opening chapter of what could become a global commercial story, contingent on PREVAIL.

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