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The F.D.A. seemed poised to rescind approval of Makena, after studies over time indicated the treatment did not halt early childbirth for many women.
The company that makes Makena, the only drug aimed at preventing preterm birth, announced on Tuesday that it was voluntarily pulling the medication off the market after advisers for the Food and Drug Administration concluded that the treatment did not help pregnant women at all.
Makena’s drugmaker, the Covis Pharma Group, said its decision had been made in deference to an F.D.A. advisory committee that agreed unanimously in October that a large study had showed that the drug offered no benefit to newborns.
Makena had been cited by critics as a flawed example of the F.D.A.’s accelerated drug approval program because the agency’s original green light for sale was based on indications that the drug would be effective. But a succession of manufacturers could not provide convincing proof after years of study that the drug halted sometimes dangerous preterm births.
Makena is now owned by Covis Pharma Group, a private-equity-backed company based in Switzerland.
“While we stand by Makena’s favorable benefit-risk profile, including its efficacy in women at highest risk of preterm birth, we are seeking to voluntarily withdraw the product and work with the F.D.A. to effectuate an orderly wind-down,” Raghav Chari, chief innovation officer at Covis, said.
The drug’s removal means that many women who have had an early birth will have no evidence-backed therapy to use during another pregnancy. While the drug was criticized for giving women false hope, patients and doctors who favored further study in the highest-risk populations spoke up in its defense at recent agency meetings.
Despite dismal study results of late, Makena was the only resort for a health risk that disproportionately affects Black women and children who have higher risks of disability or death with premature birth. The initial study of the drug that led to its accelerated approval in 2011 showed signs of promise, but a far larger trial that concluded in 2019 showed no benefit for mothers or babies.
The road to removing the drug from the market has been lengthy. The F.D.A. first proposed taking the drug off the market in October 2020. The drug’s sponsor appealed the decision, setting up a lengthy process leading to a hearing last fall.
By October of last year, 15 F.D.A. advisers voted unanimously that the lengthy so-called confirmatory study had showed no benefit to babies. All but one agreed that the drug should be withdrawn from the market.
Covis’s decision on Tuesday followed the recommendation made this past January by Dr. Celia Witten, an agency official and the presiding officer at the October hearing, that the drug be removed from the market. Still, Dr. Witten said she agreed with an advisory panel member who had acknowledged that officials could feel an imperative to “do something” when faced with a patient in need.
“I think that when we leave something on the market that hasn’t been shown to be effective, we lose out on other investigations that might be pursued,” Dr. Anjali Kaimal, an obstetrician and administrator at the University of South Florida, said during the October hearing. “And the last thing I would say is that, again, faced with that powerless feeling: Is false hope really any hope at all?”
In its news release on Tuesday, Covis said it had outlined a plan for voluntary withdrawal that included a wind-down period allowing patients using the medication to complete their courses and for the company to use its remaining inventory.
But the F.D.A. “was not in agreement with the proposal,” Covis said, and let the process advance to Dr. Witten’s recommendation.
The F.D.A.’s “accelerated approval” program is intended to grant rapid approval to a drug targeted at a serious, unmet medical need if it shows promise in delivering a benefit to patients. The program has sped about 300 drugs to the market in 30 years. It drew fierce criticism over the approval of the Alzheimer’s drug Aduhelm, an expensive drug that many experts criticized as risky and ineffective.
Congressional efforts to alter the accelerated approval process culminated last year with minor changes, including the speeding up of follow-up studies to confirm whether a drug benefits patients.
The F.D.A. should seek even more authority to improve the program, said Dr. Michael Carome, director of health research at Public Citizen, a consumer advocacy group. He said the agency advisers should be reviewing a drug seeking fast-tracked approval before an initial OK is granted. The F.D.A. should also seek authority to pull a drug from the market rapidly when the follow-up study shows no benefit, Dr. Carome said.
“Makena is a classic example,” he said, “where the clock has dragged out too long.”