The Makena controversy puts FDA’s accelerated drug approval program under

The debate over the effectiveness of a preterm labor drug highlights frustrations with FDA’s fast-track program

The Food and Drug Administration advisers’ recommendation to withdraw a preterm pregnancy drug from the market put a spotlight on the program used to approve it. 

The 14-1 vote came at the end of an emotional three-day hearing over whether the FDA should rescind approval of the drug Makena, which was initially approved through a fast-track process but whose effectiveness hasn’t been confirmed in follow-up studies, Ariana Eunjung Cha and I reported yesterday.

The debate over the drug’s effectiveness over a decade after its approval raises questions about the FDA’s accelerated approval program and underscores just how long it can take the agency to pull a drug from the market even when officials believe it doesn’t work.

In 2011, Makena was approved under the FDA’s fast-tracked process meant to expedite the availability of drugs that treat serious conditions in need of more treatments. But such approval requires drug companies to conduct follow-up studies to confirm the medicine’s benefits. 

Lawmakers on Capitol Hill, experts and even top FDA officials have said they want reforms to the program, which critics say is cumbersome and doesn’t incentivize drugmakers to perform additional clinical trials on time. 

Makena’s original trial was viewed as promising, but not definitive. However, a larger confirmatory trial out in 2019 was broadly disappointing and didn’t show the drug was effective, Ariana noted earlier this week.

The FDA’s Center for Drug Evaluation and Research proposed withdrawing it from the market in October 2020 — a move that followed an expert advisory panel’s 9-7 vote a year earlier to recommend yanking the treatment. But regulatory requirements, as well as the pandemic, slowed the process.

But drugmaker Covis Pharma, in pushing back against FDA, has argued there is evidence to suggest Makena may work in a narrower population that includes Black women at high risk of giving birth too soon. The company suggested the larger study may have missed benefits in high-risk patients in the United States because participants in the trial were mostly Eastern European, only 7 percent Black and lower risk.

That didn’t sway the FDA. Throughout this week’s meeting, top officials were steadfast in their opinion that the drug should be removed. The agency’s advisers agreed, saying it was imperative to withdraw drugs without a proven benefit from the market or risk setting a bad precedent.

Now, it’s up to FDA Commissioner Robert Califf and the agency’s chief scientist to make the final decision on what to do with the drug.

Pink Sheet’s Sarah Karlin-Smith: