Merck Lipfendra Cholesterol Pill Becomes First Oral Pcsk9 Drug Approved in US

US regulators have approved Lipfendra, a once-daily tablet that cut LDL cholesterol by more than half in late-stage trials. However, the decision does not yet make the medicine available to patients in London or elsewhere in the UK.

The US Food and Drug Administration has approved Merck’s Lipfendra cholesterol pill, making it the first oral medicine authorised to lower “bad” cholesterol by blocking the PCSK9 protein.

Lipfendra, also known by its generic name enlicitide, was approved on 16 July 2026 for adults with high cholesterol, including those with heterozygous familial hypercholesterolaemia, an inherited condition that causes persistently raised cholesterol levels. It is intended to be used alongside diet and exercise.

The decision is significant because approved PCSK9 inhibitors have previously been administered by injection. These treatments help the liver remove more low-density lipoprotein cholesterol, or LDL-C, from the bloodstream by preventing PCSK9 from reducing the number of available LDL receptors.

Statins remain the standard first treatment for high cholesterol, but some patients require additional medicines because their LDL levels remain above recommended targets or they cannot tolerate an adequate statin dose.

Trials Found LDL Reductions of Up to 59%

The FDA based its decision on two randomised, placebo-controlled Phase 3 studies involving 3,207 adults who needed further cholesterol reduction despite taking the maximum statin therapy they could tolerate.

In the larger CORALreef Lipids trial, which included 2,904 people with cardiovascular disease or an increased risk of developing it, Lipfendra reduced LDL cholesterol by 56% compared with placebo after 24 weeks.

A separate study involving 303 adults with heterozygous familial hypercholesterolaemia recorded a 59% placebo-adjusted reduction over the same period.

Those findings demonstrate the drug’s ability to lower cholesterol, but they do not yet prove that Lipfendra itself prevents heart attacks, strokes or cardiovascular deaths. Merck said a large outcomes trial involving more than 14,500 participants is continuing to examine those questions.

The approved US dose is one 20mg tablet each morning. The prescribing instructions require patients to take it on an empty stomach with water, black coffee or plain tea, then wait at least 30 minutes before eating or consuming other drinks. The tablet must be swallowed whole.

In the larger trial, adverse-reaction rates were similar among patients receiving Lipfendra and those given a placebo. In the inherited-cholesterol study, dizziness was reported in 9% of Lipfendra patients compared with 4% on placebo, while diarrhoea affected 7% compared with 2%. The US label lists no formal contraindications, although it contains specific advice concerning pregnancy.

What Does the Approval Mean for London Patients?

The FDA decision applies to the United States and does not automatically permit Lipfendra to be marketed or routinely prescribed in Britain. New medicines require a UK marketing authorisation through the Medicines and Healthcare products Regulatory Agency before entering the domestic market.

Merck, which operates as MSD outside the US and Canada, did not provide a British launch timetable in its approval announcement. Any future NHS use would also depend on UK regulatory clearance and subsequent assessment of clinical benefit and cost-effectiveness.

In the US, Lipfendra is expected to become available within weeks at a list price of $10.50 a day, or about $315 for a month’s supply, although insured patients may pay less.

For London residents following the development, the immediate change is therefore scientific rather than practical: an injection-only cholesterol-lowering mechanism is now available as a tablet in the US. The next decisive evidence will be whether ongoing research shows that the substantial LDL reductions translate into fewer major cardiovascular events.