RANKIN CO., Miss. (WLBT) – The U.S. Food and Drug Administration’s denial of a life-changing medication pushed a Rankin County family to visit Washington, D.C., and prove its effectiveness.
At the end of September, the medication for a teenager’s rare disease would no longer have been available.
However, the family’s battle for access made a change.
“Now I can actually have a life where, before, I couldn’t,” Christopher Pena said.
Pena has Barth syndrome, a rare genetic mitochondrial disease.
The teen said he is now looking forward to a normal life – walking without tiring.
Just days before the 17-year-old would not have access to Elamipretide, the FDA approved the medication.
In August, his mother, Kristi Pena, and aunt, Amy Wilson, met FDA Commissioner Dr. Marty Makary.
On Friday, they got the call that the drug was approved.
“It’s a medicine we’ve been fighting for since I was six, seven years old,” Christopher Pena said. “Knowing we have it now is amazing.”
His mother was told the FDA had until February to decide if production would be permanently halted.
“What has transpired in the past month has ultimately changed the course of history for a lot of people,” said Kristi Pena. “This is setting a precedent. This is paving the way for more rare disease developments. This highlighted and underscored a broken system, and our administration has acknowledged that.”
“I’m not worried about having to bury my son,” Amy Wilson added.
Amy Wilson’s son, Jacob, also has Barth syndrome. He was recently granted emergency access and is no longer in kidney failure.
“He has gained 10 plus pounds. His kidney function is getting better,” Wilson explained. “We know that this drug works. We know that it’s safe.”
Elamipretide, under the prescription Forzinty, is now available to around 160 people in the U.S. with certain rare mitochondrial diseases.
Approximately 300 people around the world are diagnosed with Barth syndrome.
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