An FDA advisory committee has recommended that the pregnancy drug Makena be removed from the market, indicating that the treatment designed to prevent premature birth treatment, which has become a poster child for inflated drug prices, doesn’t appear to actually work.
On October 29, a panel of experts that sit on the FDA’s Bone, Reproductive and Urologic Drugs Advisory Committee held a hearing to discuss the efficacy of Makena, which was brought into question by a recent clinical trial, which failed to find the drug was any more effective at preventing premature births than a sugar pill.
After reviewing the available data, the committee voted 9-7 to recommend that the FDA remove Makena from market by withdrawing approval for the treatment.
Makena (hydroxyprogesterone) is an AMAG Pharmaceuticals drug, which was approved by the FDA in 2011 for prevention of preterm birth in women who had a prior spontaneous preterm birth. It is the only medication approved for this use.
The drug and its original manufacturer, KV Pharmaceuticals, gained national notoriety in 2011, after the company acquired the medication that was once sold by compound pharmacies for $20, and then started charging women $1,500 per injection. The FDA eventually allowed compounding pharmacies to keep selling it to get the prices lower following public outrage.
Amag Pharmaceuticals acquired the rights to the drug in 2014. In a press release following the hearing, Amag said it was disappointed in the advisory committee’s decision.
“Preterm birth is an urgent public health crisis and the implications of leaving pregnant women and providers without access to therapy that is manufactured in a safe and regulated way is profoundly troubling,” Dr. Julie Krop, chief medical officer at AMAG, said in the press release. “For more than a decade, healthcare providers have relied on hydroxyprogesterone caproate to reduce preterm delivery in high-risk patients, which aligns with recently updated treatment recommendations of the American College of Obstetricians and Gynecologists, as well as the Society for Maternal-Fetal Medicine.”
However, the results of clinical trial, Progestin’s Role in Optimizing Neonatal Gestation (PROLONG) indicates Makena simply does not work; failing to show any clinical benefits.
The advisory committee’s recommendations are not binding, however they have a significant influence on the agency’s final ruling.
In early October, the consumer watchdog group Public Citizen called for a Makena recall in a petition to the agency, saying it did not make sense for the synthetic hormone to remain on the market to be injected into pregnant women, who would then face the risk of side effects without any apparent benefits.