The mix-up behind a controversial drug for preterm birth

Last year, the FDA withdrew its approval of Makena, the progesterone-based drug meant to prevent preterm birth, when follow-on studies failed to prove its efficacy. A little-known facet to the Makena story is largely untold, as STAT's Annalisa Merelli writes, describing a key error in the widely cited study that underpinned the drug's initial approval.

The study mixed up the names of two distinct molecules — 17P and progesterone — and this confusion persisted in dozens of papers thereafter. This led to many women being mistakenly given high doses of progesterone, which remained an untested treatment. And it took sleuth work from lab manager Tara Skopelitis, anxious to determine the cause of her daughter's unexplained illness, to recognize that 17P and progesterone were being wrongly conflated in medical literature.

17P is actually a progestin, which is a synthetic progesterone metabolite. Progesterone, meanwhile, is a naturally occurring hormone produced in the body; it can be manufactured in the lab. No study has ever shown the two molecules are interchangeable.

antibiotics

Study: Newer antibiotics are rarely used when needed

There's been a wave of newly approved antibiotics in recent years, offering an alternative to older, generic medicines that are more vulnerable to microbial resistance. But doctors aren't often using them, a new Annals of Internal Medicine analysis shows.

More than 40% of patients contending with resilient infections are still being given the older drugs, even when 80% of the time they were known to either be highly toxic or ineffective.

"There is an urgent need to understand why clinicians at hospitals with access to newer agents do not always prefer newer agents," the researchers write. "There is a stark imbalance between available new antibiotics and unmet pathogen targets… These findings call for paradigm-changing countermeasures."

regulatory

A Duchenne mom's case for accelerated approval

For families of children with Duchenne muscular dystrophy, regulatory flexibility is important in order to grease the innovation engine. A mother whose son was helped inordinately by the Duchenne gene therapy eteplirsen opines that the FDA's accelerated approval pathway needs to stay strong, so that companies remain incentivized to pursue research. The accelerated approval process, she writes, "is rooted in logic and flexibility," and helps people with no other options have a chance at a better life.

"Families know what those added years represent: more celebrations, more laughs, more stories," she writes for STAT. "More time on this planet. I can only imagine what 50 or 100 times more dystrophin could do."

obesity

Novo Nordisk's growth plan

Novo Nordisk hasn't been able to keep up with the demand for Ozempic and Wegovy, and it's taken the company a bit by surprise. As CEO Lars Fruergaard Jorgensen told the New York Times, "nobody had forecast this growth — no analyst, nobody in the company… nobody forecast a 100-year-old company would grow more than 30 percent."

The company produces more than half the world's insulin for people with diabetes, and now it has an even wider net: The obesity market is made up of nearly a billion potential patients. So the Danish company is making major changes, growing its footprint to keep up with the demand — and to stay ahead of the competition.

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