WASHINGTON, D.C. – The U.S. Food and Drug Administration (FDA) must remove from the market Makena (hydroxyprogesterone caproate) because it is not effective for preventing preterm birth or related complications, according to testimony by Dr. Michael Carome, director of Public Citizen’s Health Research Group. Dr. Carome is speaking today during the FDA’s three-day hearing to consider the agency’s proposal to withdraw approval of the drug.
The FDA approved Makena under the accelerated approval pathway in 2011, solely to reduce the risk of preterm birth in certain high-risk women. However, Makena’s approval was based largely on evidence from a single clinical trial that was seriously flawed and did not provide any evidence that the medication reduced the rate of fetal and neonatal health problems and death.
The FDA mandated that Makena’s manufacturer conduct a large, randomized post-approval clinical trial to confirm that the drug provides meaningful clinical benefits. That trial, which was completed in 2019, found that the drug is not effective for preventing preterm birth or major complications related to preterm birth. Public Citizen subsequently petitioned the FDA in 2019 to ban the drug.
In his testimony, Dr. Carome will highlight the significant flaws and limitations of the initial clinical trial supporting approval of Makena and explain why it failed to provide substantial evidence of effectiveness. He will emphasize that it is inconceivable that the FDA would have approved Makena if the data from the postmarket trial showing no benefit had been available prior to approval.
“Makena should have been removed from the market soon after the results of the post-approval trial were available,” said Dr. Carome. “The yearslong delay in the FDA withdrawing approval of the drug demonstrates fundamental deficiencies in the current regulatory oversight for drugs approved under the accelerated approval pathway. Failure to take such action now would further erode FDA’s credibility and public confidence in the agency’s accelerated approval process.”