The FDA has approved MSD's Lipfendra enlicitide as the first oral PCSK9 inhibitor for adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia. Clinical trials showed LDL-C reductions of 56% in ASCVD patients and 59% in HeFH patients. The approval marks a shift from…
The US Food and Drug Administration has granted approval to Merck and Co's Lipfendra, known generically as enlicitide, as the first oral inhibitor of proprotein convertase subtilisin/kexin type 9 PCSK9 . This macrocyclic peptide therapy offers the first non-injectable PCSK9 inhibitor for patients with cardiovascular diseases.
Lipfendra is intended to be used alongside a diet and exercise regimen. It provides an additional treatment option for adults with hypercholesterolemia, including those with heterozygous familial hypercholesterolemia HeFH . The once-daily therapy represents a significant advancement in the treatment landscape for high cholesterol, which has traditionally relied on injectable PCSK9 inhibitors.
Dr Michael Davis, Acting Director of the FDA's Center for Drug Evaluation and Research, commented on the approval. He stated that Lipfendra "provides an additional treatment option for adults with hypercholesterolemia and reflects the FDA's broader commitment to supporting meaningful advances in patient access and tackling chronic diseases." This quote underscores the agency's focus on expanding treatment choices for chronic conditions.
The FDA's decision was based on clinical data demonstrating that Lipfendra significantly reduced LDL cholesterol levels in patients with hypercholesterolemia and atherosclerotic cardiovascular disease ASCVD . In the first trial, which enrolled patients with ASCVD or those at high risk for ASCVD, Lipfendra reduced LDL-C levels by an average of 56 percent at Week 24 compared with placebo.
The second trial focused on patients with HeFH. In that study, Lipfendra enabled an average reduction of 59 percent in LDL-C levels. These results highlight the drug's efficacy across different patient populations with hypercholesterolemia.
Several other notable developments in cardiovascular and peptide therapies have occurred recently. Earlier this year, the FDA authorized AstraZeneca's Baxfendy baxdrostat as the first aldosterone synthase inhibitor ASI . This drug is intended for use in combination with other antihypertensive medications for adults with hypertension.
Separately, AstraZeneca continues to advance its own oral PCSK9 inhibitor, laroprovstat, which is currently in Phase III development. This indicates ongoing competition in the oral PCSK9 inhibitor space.
In the broader peptide therapy arena, Samsung Biologics made a significant move this week by agreeing to acquire PolyPeptide Group AG for $1.8 billion. This acquisition comes as GLP-1 peptide therapies continue to dominate market share in the pharmaceutical industry.
Additionally, the FDA approved Johnson & Johnson's Icotyde icotrokinra back in March. Icotyde is the first targeted oral peptide that precisely blocks the IL-23 receptor. The therapy is indicated for plaque psoriasis and delivers complete skin clearance alongside a favorable safety profile in a once-daily pill.
These approvals and acquisitions reflect a dynamic period in cardiovascular and peptide drug development, with multiple new oral options becoming available to patients.
Peptides referenced: GLP-1.
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