The Food and Drug Administration has removed its approval of the controversial preterm birth drug Makena, issuing Thursday a final decision that denied a request by the treatment’s maker to delay when a withdrawal would become effective.
The decision, issued by FDA Commissioner Robert Califf and Chief Scientist Namandjé Bumpus, concludes a yearslong battle between the agency and Makena’s owners over the drug’s continued use. Approved in 2011, Makena has been the only available treatment for the prevention of early labor, which is linked to a higher risk of death and disability for newborns.
But a 2019 study meant to confirm Makena’s effectiveness did the opposite, showing the drug to be ineffective and precipitating a drawn-out debate over whether there were any specific groups of women who might still benefit. FDA staff pushed for the drug’s approval to be withdrawn and independent experts convened by the agency twice endorsed removing it from market, most recently in October.
Covis Pharma, the company that sells Makena in the U.S., agreed in March to voluntarily withdraw the drug. But it asked for time to complete an “orderly wind down,” allowing women receiving Makena to finish treatment and for current stocks of the drug to be exhausted.
Califf and Bumpus declined to do so, writing in their decision memo that “there is an insufficient demonstration of effectiveness to balance any level of risk.”
“Without a favorable benefit-risk assessment, there is no justification for keeping the product on the market, even where there is an unmet need,” they wrote.
However, they left implementation of their order to the FDA’s Center for Drug Evaluation and Research, which could still choose to permit patients to complete their treatment course.
They also deferred to CDER on how to address compounding of the active drug ingredient in Makena. The main hormone used in Makena, hydroxyprogesterone caproate, was available via compounding pharmacies prior to the drug’s 2011 approval and Covis has argued patients will again turn to this alternative following a withdrawal.
Apart from compounded versions, women at high risk of preterm birth will now have no treatment options, a state of affairs Covis highlighted in making the case for its drug’s continued availability.
Califf and Bumpus acknowledged those concerns in their decision, urging further research on new medicines.
“It is tragic that the scientific research and medical communities have not yet found a treatment shown to be effective in preventing preterm birth and improving neonatal outcomes,” they wrote. “Nothing in this opinion today is intended to minimize these concerns — to the contrary, our hope is that this decision will help galvanize further research.”