FDA panel backs removal of unproven pregnancy drug

WASHINGTON (AP) — Federal health advisers have concluded that a drug intended to prevent preterm birth has not been shown to work, clearing the way for U.S. regulators to press ahead with a long-delayed effort to pull it. of the market.

WASHINGTON (AP) — Federal health advisers have concluded that a drug intended to prevent preterm birth has not been shown to work, clearing the way for U.S. regulators to press ahead with a long-delayed effort to pull it. of the market.

The Food and Drug Administration’s advisory committee voted 14 to 1 on Wednesday to withdraw the injectable drug Makena, despite pleas from the manufacturer to keep it available pending further research.

Experts essentially agreed with a 2020 FDA ruling that deemed the drug ineffective and called for its withdrawal. The manufacturer, Covis Pharma, had challenged that decision, putting in place this week rare public hearing.

“If we allow Makena to remain on the market, it implies that the FDA reviewed a large study, found no benefit, and yet allowed this drug to remain on the market,” said Dr. Margery Gass, obstetrics specialist. “I think it’s a bad precedent.

Gass and other panelists encouraged Covis to continue studying the drug to determine if a subset of women might benefit from it.

“I think our patients deserve a response and they deserve this well-designed clinical trial and I think taking the drug off the market is going to enable that,” said Dr. Mary Munn of the University of South Alabama.

FDA Commissioner Dr. Robert Califf is expected to make a final decision on withdrawing the drug in the coming months. If he follows the panel’s advice, it would be the first time the FDA has officially withdrawn a drug it originally approved based on promising early data.

The FDA granted accelerated approval to Makena in 2011 based on a small study in which it appeared to reduce the rate of preterm birth in women with a history of the problem. Premature birth increases the risk of disability and death in infants and affects approximately 10% of deliveries in the United States.

FDA approval was conditional on a larger follow-up study to confirm whether Makena resulted in healthier outcomes for babies.

But in 2019, results from this international study of 1,700 patients showed that the drug did not reduce preterm births – as originally thought – or lead to healthier outcomes for infants.

The FDA has since decided to revoke the drug’s approval. But the lengthy bureaucratic process underscores the difficulty of removing a drug from the market when a manufacturer does not do so voluntarily.

Luxembourg-based Covis Pharma argued this week that Makena benefits women most at risk of early childbirth, including black Americans, and offered to restrict its approval to this group while it conducts another study to confirm. its efficiency. The company’s presentations included outside experts who said removing Makena would worsen racial disparities in prenatal care.

“The FDA should be doing what’s best for our patients, which is to keep this drug available to those of us who manage these very high-risk patients on a daily basis,” Mr. FDA Dr. Yolanda Lawson, an obstetrics specialist at Baylor University. sign.

But the experts ultimately sided with FDA scientists, who pointed out that they were “unable to identify a group of women for whom Makena had an effect.” The drug also carries risks, the FDA noted, including blood clots and depression.

The FDA has come under pressure to crack down on unproven drugs approved under its accelerated approval programwhich has launched dozens of drugs based on early results since the early 1990s. The flip side of the program is to remove drugs if their initial promise is not confirmed by subsequent studies.

Makena has become something of a poster child for the program’s downsides, as the drug has remained on the market for over a decade without any confirmed benefits. According to a recent federal report, the United States has spent $700 million on Makena since 2018 through various government programs, including Medicaid.

About 350,000 women have taken the drug over the past decade, according to Covis.

Even if the drug is withdrawn, some doctors are expected to continue prescribing its key ingredient as a specialty drug. Preparation pharmacies have long offered their own formulations of the drug. Makena and compound drugs consist of a synthetic form of the hormone progesterone, which helps the uterus grow and maintain pregnancy.


This story has been corrected to show that the panel member who commented on the potential bad precedent was Margery Gass, not Margaret Gatz.


The Associated Press Health and Science Department is supported by the Howard Hughes Medical Institute Department of Science Education. The AP is solely responsible for all content.

Matthew Perrone, The Associated Press

Carol N. Valencia