The only drug on the market used to prevent premature births could soon be pulled from shelves as regulators fear it does not work.
The Food and Drug Administration (FDA) declared the drug Makena, produced by the Swiss firm Covis Pharma, ineffective two years ago.
A three-day hearing is now underway, as the company is challenging the agency’s declaration and hoping to avoid the drug’s removal from the market.
Its approval came under the agency’s accelerated approval program in 2011 after a trial showed it could prevent premature birth in women who had a history of such.
The drug has since been put under more scrutiny. In recent years, the FDA found that repeated injections of Makena showed little effect on premature birth rates.
A 2019 study found that its use did not lead to reduced premature birth rates or healthier outcomes for the infant.
The FDA wants it pulled as it could provide false hope to some mothers and also lead to resources that could be invested elsewhere going into a dead-end treatment.
Complicating the Makena debate is support from the leading U.S. obstetrics group to keep the decade-old drug available while more research is done.
About 10 per cent of US births come too early — before 37 weeks, raising the risk of serious health problems and even death in infants.
The injectable drug Makena may soon be pulled from US markets. The FDA is holding hearings to decide the fate of the drug this week after a 2019 study found that it did little to prevent premature births or lead to better health for the baby when it was born
The hearing will resemble a courtroom trial, with FDA staff and company scientists presenting arguments for and against the drug Makena.
There will be a vote Wednesday by a panel of outside experts on the fate of the drug.
Agency leaders will ultimately make the final decision whether to order a withdrawal.
‘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.’
The FDA warns that the drug could be providing little for mothers using it hoping it would boost their pregnancy health.
‘Based on the evidence shown today, Makena is not shown to be effective,’ said FDA’s drug chief, Dr Patrizia Cavazzoni, in opening remarks Monday.
‘Its benefit and risk profile is unfavorable and it should be withdrawn from the market.’
Around 10 per cent of births in the US are pre-term. The FDA warns that Makena gives many expecting mothers ‘false hopes’ (file photo)
Premature birth – and the risks it poses to babies
Around 10 per cent of all pregnancies worldwide result in premature labor – defined as a delivery before 37 weeks.
When this happens, not all of the baby’s organs, including the heart and lungs, will have developed. They can also be underweight and smaller.
Tommy’s, a charity in the UK, says this can mean preemies ‘are not ready for life outside the womb’.
Premature birth is the largest cause of neonatal mortality in the US and the UK, according to figures.
Babies born early account for around 1,500 deaths each year in the UK. In the US, premature birth and its complications account for 17 per cent of infant deaths.
Babies born prematurely are often whisked away to neonatal intensive care units, where they are looked after around-the-clock.
What are the chances of survival?
- Less than 22 weeks is close to zero chance of survival
- 22 weeks is around 10%
- 24 weeks is around 60%
- 27 weeks is around 89%
- 31 weeks is around 95%
- 34 weeks is equivalent to a baby born at full term
The dispute is likely to increase scrutiny of the agency’s so-called accelerated approval program.
It allows drugs like Makena to launch based on promising early results while additional, usually larger, studies are conducted.
‘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 of 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 FDA approved Makena in 2011 based on a small study suggesting it reduced rates of premature birth in women with a history of early deliveries.
Makena consists of a synthetic version of the hormone progesterone, which helps the uterus grow and maintain a pregnancy.
Women can start the shots after 16 weeks of pregnancy.
In briefing documents released this month, the FDA said leaving Makena on the market ‘incurs false hopes, the risks associated with treatment, and other burdens’ like excess medical spending.
According to a recent federal report, the US has spent $700 million on Makena since 2018 through various government programs, like Medicaid and Veterans Affairs.
The FDA opinion also applies to several generic versions of the shot.
The drugmaker argues that Makena is effective and that flaws in the 2019 study obscured its benefit.
The company points out that Black women are at higher risk for preterm birth, but made up just 7 per cent of women in the international study, compared with 59 per cent in the original U.S. study used for approval. It wants time to do another study in higher risk patients, particularly Black Americans.
A patient group set up with funding by Covis, the Preterm Birth Prevention Alliance, has made similar arguments in letters to the FDA.
‘We believe there is no sound public health reason to deprive’ patients and physicians of access to Makena, the company said in an emailed statement.