Amgen's Repatha trial shows heart risk reduction

Amgen said yesterday that its cholesterol-lowering injection Repatha cut the risk of a composite of heart disease death, heart attacks, and strokes in a 12,000-patient trial, bolstering hopes for wider use of PCSK9 inhibitors after years of slow adoption, STAT's Matt Herper writes.

The drug, which brought in $2.2 billion in 2024 sales, met both main goals in the VESALIUS-CV study, though full data aren't yet available. Repatha has been available since 2015, but these new results could grow its market.

"Amgen has indicated that current penetration remains in the low single digits, suggesting the near-term opportunity lies in increasing urgency to treat rather than expanding the patient pool," one Leerink analyst said after previewing the results in August.

DRUG PRICING

CMS delays effort to address IRA loophole

The Trump administration is delaying a proposal to crack down on a loophole that allows drugmakers to avoid Medicare price negotiation on some of their products by making minor tweaks.

The Inflation Reduction Act currently allows drugmakers to avoid price negotiations by combining ingredients. In draft guidance earlier this year, Medicare officials said they were trying to figure out how to handle products that combine an active ingredient subject to negotiation with an ingredient that does not result in clinically meaningful difference.

But the final version of that guidance, published this week, states that the Centers for Medicare & Medicaid Services is putting off that policy for another year, STAT's John Wilkerson writes.

abortion

FDA approves new generic version of mifepristone

The FDA has approved a second generic version of the abortion pill mifepristone — a low-cost copy made by Evita Solutions. The decision to approve the drug, nearly four years after the company applied, drew swift backlash from anti-abortion groups and Republican lawmakers, including Sen. Josh Hawley, who blasted the decision as a failure of FDA leadership.

The approval comes as Trump administration health officials, led by HHS Secretary Robert F. Kennedy Jr. and FDA Commissioner Marty Makary, face mounting pressure from abortion opponents to revisit the drug's safety, despite 25 years of FDA data affirming its safety and effectiveness. Access to mifepristone, used with misoprostol in two-thirds of U.S. abortions, remains sharply limited in states with abortion bans.

podcast

A pharma C-suite shakeup and Trump's deal with Pfizer

How will we look back on Emma Walmsley's tenure as GSK's CEO? Why did one of the FDA's top drug officials call out an obscure lupus drug on LinkedIn? And will we ever stop talking about the pharma patent cliff?

We discuss all that and more on this week's episode of "The Readout LOUD," a weekly biotech podcast from STAT.

Adam is out this week, so Allison and Elaine dive into the latest news, starting with another biotech acquisition and a new CEO at GSK. Then, they invite health policy researcher Stacie Dusetzina to break down President Trump's "most-favored nation" pricing deal with Pfizer.

Listen here.

animal research

NIH's Nicole Kleinsteuer pushing past animal research

Nicole Kleinstreuer, a newly appointed acting deputy director at NIH, is spearheading the creation of a new office — the Office of Research, Innovation, Validation, and Application — to accelerate "new approach methodologies" that reduce reliance on animal testing. A math nerd turned biomedical engineer, Kleinstreuer has long argued that outdated animal models contribute to drug failures in humans, and she envisions flipping the paradigm so animal studies become the "alternative."

"I would love for us to advance the science so dramatically and so comprehensively and build out this amazing toolbox of technologies and cutting-edge approaches," Kleinsteuer told STAT contributor Sara Talpos.

ORIVA aims to fund organoid research and prioritize human-based models. Some scientists worry that it's too early to pivot away from animal research, though Kleinstreuer has said she won't phase out animal studies overnight — which actually brought on attacks from animal activists. Her idea, however, is to push forward with for faster, more predictive, and less animal-dependent science.

More around STAT