RANKIN CO., Miss. (WLBT) – A Rankin County teenager’s life-changing medication is being denied approval by the U.S. Food and Drug Administration, leaving him and many others with rare diseases unsure of their futures.
According to the teen’s family, the medication is safe and they blame red tape for the pharmaceutical company being forced to halt production.
“It made it easier to have a life,” Christopher Pena said.
The denial of this medication could result in Pena’s restored health declining once again. The 17-year-old has Barth Syndrome, a rare metabolic disease that impacts both the muscles and immune system.
The FDA recently denied approval of the drug Pena takes for his condition, called Elamipretide.
“I was able to actually start going to do things with my friends and hang out with them,” Pena said. “I could go fishing or hunting where before I could barely go 30 feet without having to stop because of the fact I was too tired.”
On Monday, his mother, Kristi Pena, learned that Stealth BioTherapeutics, the pharmaceutical company which produces the drug, would be closing its doors.
The organization invested $100 million into the medication’s development and, after the FDA’s decision, investors withdrew their support.
“I’m not saying that this drug is a cure or that it is God, but I am saying that it is life-changing and we deserve a chance to have access to it,” Kristi Pena said. “It is safe. It is effective. There is zero reason for the FDA to tell us no. We have dotted every I. We have crossed every singe T.”
Christopher’s first cousin, Jacob Wilson, also has Barth Syndrome and was taken off the medication.
At 24-years-old, he weighs 70 pounds, is connected to a feeding tube, and has kidney decline.
His mother, Amy Wilson, said she believes the lives of those suffering do not matter to the FDA.
“The only hope that we have is Commissioner Makary, RFK Jr., or President Trump himself stepping in and saying this drug is effective. It’s not a safety issue. It’s not an efficacy issue. It’s red tape,” said Amy Wilson.
By the end of August, Christopher Pena and others with similar rare diseases will no longer have access to the medication they need.
“It’s not a matter of ‘Is this disease going to kill my son.’ It’s a matter of when,” Kristi Pena said. “We’re just not going to give up.”
These families are continuing to reach out to the Mississippi Congressional Delegation and say they are considering a trip to Washington, D.C. for a peaceful protest.
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