Merck's Lipfendra, the first PCSK9-targeting pill, cut LDL by 55% in a key trial. The approval challenges Amgen's injection franchise and could expand the addressable patient pool.
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WASHINGTON – The FDA approved Merck's Lipfendra on Thursday, the first pill that blocks the liver protein PCSK9 – a mechanism that has been available only through injectable drugs for more than a decade. The drug is cleared for patients whose LDL cholesterol stays high even after maximum statin therapy.
Merck won the approval on two late-stage studies. In a 3,000-patient trial, those who added Lipfendra to statins saw LDL drop more than 55% after six months. A second study showed a 59% average reduction versus placebo. The benefit held over a year, and side effects – dizziness and diarrhea – were similar between the drug and placebo groups, researchers found. The pill must be taken on an empty stomach.
The approval gives Merck a direct shot at a market that Amgen has dominated since 2015 with its injectable Repatha. Novartis also competes with Leqvio, a twice-yearly shot that hit $2.3 billion in sales last year. The injectables have struggled with patient access: high list prices, insurance restrictions, and a narrow prescribing base limited uptake. An oral version could expand the pool of eligible patients, especially those who avoid injections.
Heart disease is the leading cause of death in the U.S., and high LDL cholesterol is a top risk factor for heart attacks and strokes. Doctors recommend lowering LDL to at least 70 mg/dL for patients with established disease, and below 55 mg/dL for very high-risk patients. Statins remain the standard first-line treatment, blocking the liver's production of cholesterol. Even at the highest doses, many patients still need additional help meeting those targets.
Merck, based in Rahway, New Jersey, will market Lipfendra under that brand name. The FDA reviewed the drug under a fast-track program created by then-FDA chief Dr. Marty Makary, who resigned in May after months of pressure from drugmakers and patient groups. The agency cited the drug's potential to offer a public health benefit in approving it within six months of the application.
The key open question is pricing and insurance coverage. Amgen's Repatha has a list price of roughly $14,000 a year, though rebates bring net costs lower. Merck has not disclosed Lipfendra's price. Insurers have been reluctant to cover PCSK9 injectables without prior authorization, and it remains unclear whether they will treat an oral drug differently. The company will need to show that the pill's convenience outweighs the injection barrier for patients and prescribers alike.
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