FDA requires withdrawal of preterm birth prevention drug approval

Key takeaways :

• FDA withdrew approval of Makena due to lack of clinical benefit.
• No medication is currently approved to reduce preterm birth risks.

The FDA withdrew approval of hydroxyprogesterone caproate injection, the only drug that had been approved to reduce risk for preterm birth in women with a history of spontaneous preterm birth, according to an agency press release.

According to the release, benefits of hydroxyprogesterone caproate injection (Makena, Covis Pharma) do not outweigh risks associated with use.

“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 — particularly in light of the fact that this serious condition has a disparate impact on communities of color, especially Black women,” Robert M. Califf, MD, FDA Commissioner, said in the release. “Fundamentally, however, the touchstone of FDA drug approval is a favorable benefit-risk assessment; without that favorable assessment, the drug should not have the status of being FDA-approved.”

As Healio previously reported, Makena was approved under the accelerated approval pathway in 2011 based on data that was “reasonably likely to predict clinical benefit” with the drug. However, a required post-marketing confirmatory study failed to show clinical benefit. Following a hearing in October, the FDA Obstetrics, Reproductive and Urologic Drugs Advisory Committee recommended withdrawing Makena’s approval.

This final withdraw decision was issued by the FDA Commissioner and Chief Scientist due to poor findings in the review. Makena and its generics are no longer approved and cannot be distributed in interstate commerce.

“We acknowledge at the outset the serious problems of preterm birth with respect to both maternal and neonatal health and the contribution of institutional forces that have led to health disparities, including preterm birth, among Black women,” Namandjé Bumpus, PhD, chief scientist of the FDA, said. “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.”