In a three-day hearing that began Monday, a U.S. Food and Drug Administration advisory committee will give the maker of a drug meant to prevent premature births a chance to show why its drug should not be pulled off the market.
FDA officials have said they want to withdraw the medication, Makena, because of lack of evidence that it works and due to its side effects.
The drug was initially allowed as part of the agency's accelerated approval program, which lets a medication launch after promising early results while larger studies test it further.
“Based on the evidence shown today, Makena is not shown to be effective,” Dr. Patrizia Cavazzoni, director of the FDA's Center for Drug Evaluation and Research, said in opening remarks on Monday, the Associated Press reported. “Its benefit-and-risk profile is unfavorable, and it should be withdrawn from the market.”
Complicating the issue is that Makena, a weekly injectable, is the only medication that exists to help prevent preterm birth, the AP reported.
“The need for an effective treatment for preterm birth is great,” the American College of Obstetricians and Gynecologists says. “Makena and its associated generics represent the only treatment currently available to obstetrician-gynecologists to help prevent this condition.”
At the hearing, FDA staff and company scientists will each present their arguments. The FDA panel of outside experts will vote on the issue at the conclusion of the hearing on Wednesday, but the agency will still make the final decision about whether to order a withdrawal, the AP reported.
Makena has been on the marketplace since 2011, when a small study showed it reduced rates of early delivery in women with a history of early delivery, the AP reported.
But a 2019 international study of 1,700 women found the drug didn't reduce premature births but could cause side effects that included blood clots and depression.
The drugmaker, Covis, has said that later study was flawed. Only 7% of participants were Black women, who have a higher risk for preterm birth, compared to 59% that participated in the original U.S. study, the company said. Covis wants to conduct another study with higher-risk patients, especially Black Americans.
Still, leaving Makena on the market “incurs false hopes, the risks associated with treatment, and other burdens,” like excessive medical spending, the FDA said in documents posted earlier this month.
Preterm birth can lead to serious health conditions and even infant death. About 10% of U.S. births happen before 37 weeks, CDC data shows.
Makena is a synthetic version of progesterone, with shots that begin after 16 weeks of pregnancy. That hormone helps a uterus grow and maintain a pregnancy.
The battle has increased scrutiny of the FDA's accelerated approval program.
While the program has sped availability of breakthrough therapies for HIV and cancer, it has also been criticized for not following up when drugs have incomplete or unconvincing data. That includes many expensive cancer medications on the market, the AP reported.
“Makena is being used as an example of the many different criticisms of this program,” said Rachel Sachs, a food and drug law specialist at Washington University in St. Louis. “That may not be fair to the other drugs, other diseases, other patients' groups, but we're forced to respond to the situation it presents.”
The U.S. Centers for Disease Control and Prevention has more on preterm birth.
SOURCE: Associated Press