WELL, AN OMAHA MOM SAYS A NEW MEDICATION SAVED HER INFANT SON FROM NEAR CERTAIN DEATH. BUT NOW SHE FEARS SHE’LL LOSE ACCESS TO IT. AFTER THE FDA DECLINED APPROVAL OF THE DRUG LAST WEEK, KETV NEWSWATCH SEVEN’S AARON HEGARTY IS HERE IN THE STUDIO WITH THE EXCLUSIVE STORY. AARON. YEAH. ROB. JULIE, THIS IS ONE YEAR OLD JALEN. HE HAS BARR SYNDROME, A GENETIC CONDITION ESTIMATED TO IMPACT ONLY AROUND 150 PEOPLE IN THE U.S. HIS MOM SAYS A DEVELOPMENTAL DRUG DRUG TURNED TURNED HIS LIFE AROUND. BUT SHE SAYS THE FIGHT IS FAR FROM OVER. PLATTE ARE. SO WHEN YOU SEE ONE YEAR OLD JALEN NOW HE’S DOING ALL THE THINGS A ONE YEAR OLD COULD DO. YOU MIGHT NOT GUESS THAT THIS IS WHAT HE FACED WHEN HE WAS ONE DAY OLD. HE’S A FIGHTER FOR SURE. HIS MOM, JORDAN CARLEY, SAYS THE DOCTORS GAVE JALEN A 40% CHANCE OF SURVIVAL. HE HAS A RARE GENETIC DISORDER, BARTH SYNDROME, THAT IMPACTS THE HEART. I ACTUALLY GOT THE DIAGNOSIS OF HIM AT 23 WEEKS PREGNANT. I KNEW I WAS A CARRIER. IT ALMOST EXCLUSIVELY IMPACTS BOYS. JALEN HAS TWO OLDER SISTERS, BUT HE’S CARLEE’S FIRST BOY. CARLEY’S BROTHER DIED WITH BARR SYNDROME AT JUST 18 MONTHS OLD IN 2004, AND JALEN FACED A SIMILAR FATE. HIS HEART WAS FAILING FROM BIRTH. THE SURGEON ACTUALLY CAME IN THE ROOM AND TOLD ME LIKE, LOOK, DO YOU WANT TO SIGN A DNR FOR YOUR SON? HE WAS LIKE A DAY OLD AND I IMMEDIATELY REFUSED. BUT AT ONE MONTH OLD, JALEN BEGAN ALMA AVAILABLE ONLY THROUGH SPECIFIC PROGRAMS, SHE SAYS, WITH NO OTHER CHANGES. HIS HEART FUNCTION WENT FROM 20% TO NORMAL IN A MONTH. HE WAS WHAT WE PRAYED FOR WITH THIS MEDICATION AND WE GOT THAT AND WE HAVE GOTTEN THAT FOR THE PAST YEAR. BUT LAST WEEK, THE FDA DENIED AN APPLICATION FOR APPROVAL OF ALMA, ACCORDING TO THE PRODUCER OF THE DRUG AND THE BARTH SYNDROME FOUNDATION. BUT THE FDA DID PROPOSE A PATHWAY FOR ACCELERATED APPROVAL. THE FOUNDATION’S DIRECTOR CALLS THE DECISION UNCONSCIONABLE. IN A STATEMENT, CARLEY TRAVELED TO D.C. LAST YEAR AS A COMMITTEE RECOMMENDED APPROVAL ON A 10 TO 6 VOTE. SHE AND JALEN MET CONGRESSMAN MIKE FLOOD IN THE PROCESS, WHO WROTE A LETTER TO THE FDA IN MARCH CALLING FOR APPROVAL OF LM APATITE, SAYING IT ALREADY FACED AN UNUSUALLY LONG REVIEW. CARLEY SAYS APPROVAL FOR ALL AGES CAN’T WAIT, AND SHE FEARS THAT WITHOUT IT, JALEN WILL EVENTUALLY LOSE ACCESS BECAUSE PRODUCTION OF THE DRUG WOULD END. WE’VE BEEN TOLD THAT, YOU KNOW THERE’S GOING TO BE CHANGE WITHIN THE FDA FOR A RARE DISEASE COMMUNITY, AND YOU’RE NOT SHOWING THAT EITHER. AND QUITE HONESTLY, NOT APPROVING THIS MEDICATION IS A DEATH SENTENCE TO NOT JUST MY SON, BUT EVERYONE LIVING WITH IT. CARLEY SAYS. JALEN DOESN’T SEEM TO HAVE ANY SIDE EFFECTS. SHE SAYS JALEN IS A BRAVE ONE. SHE GIVES HIM A SHOT WITH THE DRUG EVERY NIGHT, BUT SHE SAYS SHE’S TOLD IF APPROVAL DOESN’T COME IN AS LITTLE AS SIX MONTHS. JALEN AND EVERYONE COULD LOSE ACCESS
As FDA delays approval, Omaha mom fears son with rare disorder could lose access to drug she says is saving his life
“Not approving this medication is a death sentence to not just my son,” an Omaha mom told KETV, “but everyone living with” Barth syndrome.
Updated: 8:32 AM CDT Jun 2, 2025
An Omaha mom says Food and Drug Administration approval of a drug intended to address her son’s rare condition can’t wait.Jordan Karle’s 1-year-old son Jaylen experienced heart failure from birth because of Barth syndrome, a deadly, rare condition estimated to affect around 150 people in the U.S.The FDA denied approval of the drug, elamipretide, last week, but gave a pathway to approval, according to the drug manufacturer and the Barth Syndrome Foundation.A day after birth, Jaylen was given a 40% chance of survival, Karle said.A month after birth in early 2024, Jaylen began taking the drug, which is available to critically ill babies through a program now, Karle said. Then, Jaylen’s heart functioned at 20%. In another month, his health functioned at a normal rate, Karle said. She said after more than a year on the drug, he is doing well and not experiencing side effects.Karle’s brother died at 18 months old with Barth syndrome in 2004, Karle said. Although the syndrome is rare, it is not in her family. A 16-year-old cousin survives with a new heart, she said. Jaylen is the first to have access to elamipretide.Karle says Barth syndrome patients of all ages deserve access to the drug. She says Jaylen is also at risk without FDA approval.”We’ve been told that there’s going to be change within the FDA for the rare disease community,” Karle said. She said the FDS is “not showing that.”She added: “Not approving this medication is a death sentence to not just my son, but everyone living with” Barth syndrome.Karle traveled to Washington, D.C., last fall to speak to a committee that would eventually recommend approval of the drug on a 10-6 vote. She also met with Congressman Mike Flood, who wrote a letter to the FDA in March. In that letter, Flood wrote that the review process for elamipretide was already unusually long.
OMAHA, Neb. —
An Omaha mom says Food and Drug Administration approval of a drug intended to address her son’s rare condition can’t wait.
Jordan Karle’s 1-year-old son Jaylen experienced heart failure from birth because of Barth syndrome, a deadly, rare condition estimated to affect around 150 people in the U.S.
The FDA denied approval of the drug, elamipretide, last week, but gave a pathway to approval, according to the drug manufacturer and the Barth Syndrome Foundation.
A day after birth, Jaylen was given a 40% chance of survival, Karle said.
A month after birth in early 2024, Jaylen began taking the drug, which is available to critically ill babies through a program now, Karle said. Then, Jaylen’s heart functioned at 20%. In another month, his health functioned at a normal rate, Karle said. She said after more than a year on the drug, he is doing well and not experiencing side effects.
Karle’s brother died at 18 months old with Barth syndrome in 2004, Karle said. Although the syndrome is rare, it is not in her family. A 16-year-old cousin survives with a new heart, she said. Jaylen is the first to have access to elamipretide.
Karle says Barth syndrome patients of all ages deserve access to the drug. She says Jaylen is also at risk without FDA approval.
“We’ve been told that there’s going to be change within the FDA for the rare disease community,” Karle said. She said the FDS is “not showing that.”
She added: “Not approving this medication is a death sentence to not just my son, but everyone living with” Barth syndrome.
Karle traveled to Washington, D.C., last fall to speak to a committee that would eventually recommend approval of the drug on a 10-6 vote. She also met with Congressman Mike Flood, who wrote a letter to the FDA in March. In that letter, Flood wrote that the review process for elamipretide was already unusually long.