HEALTH: FDA panel recommends withdrawal of preterm birth drug

An advi­so­ry pan­el to the U.S. Food and Drug Admin­is­tra­tion (FDA) on Wednes­day vot­ed to with­draw the drug from the mar­ket, cit­ing doubts about its effec­tive­ness in pre­vent­ing pre­ma­ture births.

Three days of Obstet­rics, Repro­duc­tive and Urol­o­gy Drug Advi­so­ry Board rec­om­men­da­tions for clin­i­cal tri­al evi­dence in sup­port of Mak­e­na, the only US-approved anti-preterm birth drug, come to a close with a 14-to‑1 vote I was.

There was also emo­tion­al tes­ti­mo­ny from advo­cates at the con­fer­ence that dis­con­tin­u­ing the drug could widen mater­nal health disparities.

The pan­el vot­ed on three ques­tions: whether the drug works, whether tri­al data sup­port approval, and whether the drug should remain on the mar­ket. Dur­ing the pan­el dis­cus­sion, I vot­ed against each question.

“It’s unfair to keep drugs on the mar­ket and expose par­tic­u­lar­ly vul­ner­a­ble peo­ple to inef­fec­tive treat­ments,” said Dr. Mark Had­dock, a pedi­a­tri­cian at the Uni­ver­si­ty of Flori­da School of Med­i­cine in Jacksonville.

Pre­ma­ture birth is defined as being born before 37 weeks, and about 1 in 10 births in the Unit­ed States is pre­ma­ture, accord­ing to the Cen­ters for Dis­ease Con­trol and Pre­ven­tion. Black women in the Unit­ed States are at increased risk for this com­pli­ca­tion, and their babies may have chron­ic health prob­lems through­out their lives.

Mak­e­na is a syn­thet­ic hor­mone that is usu­al­ly giv­en once a week start­ing at the 16th week of pregnancy.

Wednes­day’s rec­om­men­da­tions are based heav­i­ly on data from a 2019 clin­i­cal tri­al of more than 1,500 women who received Mak­e­na injec­tions once a week from 16 weeks’ ges­ta­tion, or a place­bo. They found that it did not reduce the risk of pre­ma­ture birth or improve the health of newborns.

The deci­sion is in the hands of the FDA, who may make a final deci­sion in the com­ing months.

How­ev­er, the agency has already once pro­posed revok­ing the drug’s approval.

The drug received accel­er­at­ed approval from the FDA in 2011 after its effi­ca­cy was con­firmed in a small clin­i­cal tri­al involv­ing more than 500 women. How­ev­er, the approval was con­tin­gent on the man­u­fac­tur­er, AMAG Phar­ma­ceu­ti­cals, con­duct­ing addi­tion­al stud­ies to show the effi­ca­cy of the drug.

The same FDA advi­so­ry pan­el vot­ed to rescind the drug after tri­als con­duct­ed in 2019 showed the drug to be unprofitable.

And a year lat­er, the FDA pro­posed remov­ing the drug from the mar­ket, but Covis Phar­ma, now the mak­ers of Mak­e­na, request­ed addi­tion­al hear­ings to make their case.

In slides released ahead of this week’s meet­ing, FDA sci­en­tists said Mak­e­na had not been shown to be effec­tive and put women at “seri­ous risks” such as blood clots, aller­gic reac­tions and depres­sion. I have not changed my posi­tion on this drug.

Addi­tion­al­ly, FDA sci­en­tists wrote, “Retain­ing approval of Mak­e­na is like­ly to ham­per research into more promis­ing treat­ments for preterm birth.”

At the three-day con­fer­ence, the com­pa­ny’s rep­re­sen­ta­tives insist­ed that its drug remains an “essen­tial tool” for pre­vent­ing pre­ma­ture births.

The com­pa­ny sug­gest­ed to pan­el mem­bers that it keep Mak­e­na on the mar­ket while the FDA con­ducts anoth­er clin­i­cal tri­al that could prove its usefulness.

He also sug­gest­ed with­draw­ing some of the drugs so that they could only be used by women at high risk of pre­ma­ture birth.

“Cor­bis is com­mit­ted to work­ing with author­i­ties to explore appro­pri­ate options for fur­ther research, includ­ing designed con­trolled clin­i­cal tri­als, obser­va­tion­al stud­ies, and reduced label­ing to focus on patients at high­est risk. ‚” the com­pa­ny said in a state­ment before the vote.