USA – Makena indication may fall based on post-approval data

The US Food and Drug Administration (FDA) is seeking to withdraw the approval of the only drug indicated to prevent recurrent preterm birth, based on a post-approval study showing a lack of efficacy.

Makena (hydroxyprogesterone caproate injection) was originally granted accelerated approval in 2001 and remains on the market. Makena’s manufacturer, AMAG Pharmaceuticals, has requested a public hearing to explore the issue. It is up to the FDA commissioner whether to grant the hearing request and ultimately whether to withdraw approval for the drug.

“We sympathize with women who are at risk for recurrent preterm birth that could result in death or significant lifelong health effects in neonates, but retaining on the market a drug not shown to be effective for this use does not protect or promote their health,” Christina Y. Chang, MD, and colleagues from the FDA Center for Drug Evaluation and Research (CDER) wrote in a perspective article published 3 November 2020 in the New England Journal of Medicine