The U.S. Food and Drug Administration has denied approval for Elamipretide, a treatment for the rare metabolic disease Barth Syndrome, forcing pharmaceutical company Stealth BioTherapeutics to cease operations and leaving patients without access to a medication they describe as life-changing.
The denial affects patients like 17-year-old Christopher Pena of Rankin County, Mississippi, who experienced dramatic improvements in his condition while taking Elamipretide. Before the treatment, Pena could "barely go 30 feet without having to stop because of the fact I was too tired." After beginning the medication in November, he gained the ability to climb stairs easily, jump with both feet leaving the ground, and participate in activities with friends including fishing and hunting.
Company Closure Following FDA Rejection
Stealth BioTherapeutics announced it would close its doors after the FDA's decision, having invested $100 million in Elamipretide's development. Following the regulatory rejection, investors withdrew their support from the company, making continued operations financially unsustainable.
"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," said Kristi Pena, Christopher's mother. "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 single T."
Impact on Barth Syndrome Patients
Barth Syndrome is a rare metabolic disease that impacts both muscles and the immune system. The condition's severity is illustrated by the case of Jacob Wilson, Christopher's 24-year-old cousin who also has Barth Syndrome. After being removed from Elamipretide treatment due to developing a minor rash during clinical trials, Wilson's health declined significantly. He now weighs 70 pounds, requires a feeding tube, and has experienced kidney function decline.
"He is not on it now. His health has declined. He now has a feeding tube," said Amy Wilson, Jacob's mother. "He's lost significant amounts of weight. His kidney function has declined to the point where he didn't even feel well enough to get out of the bed."
Patient Access Timeline
By the end of August, Christopher Pena and other patients with similar rare diseases will no longer have access to Elamipretide. The medication had enabled Christopher to transition from requiring hospice care before his first birthday to living an active teenage life, attending Brandon High School ceremonies and participating in normal activities.
Video documentation shows the dramatic difference in Christopher's mobility, with footage from before treatment showing him slowly struggling to climb stairs, while recent video demonstrates him easily walking up stairs during a school ceremony.
Advocacy Efforts and Political Appeals
Families affected by the FDA's decision are reaching out to the Mississippi Congressional Delegation and considering a peaceful protest in Washington, D.C. They are specifically seeking intervention from new FDA Commissioner Marty Makary, as well as Robert F. Kennedy Jr. and President Trump.
"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.
The families plan to meet with Senator Cindy Hyde-Smith to encourage the new FDA Commissioner to focus on approving medical treatments for patients with ultra-rare diseases. They argue that the denial represents regulatory obstacles rather than legitimate safety or efficacy concerns.
"It's not a matter of 'Is this disease going to kill my son.' It's a matter of when," said Kristi Pena. "We're just not going to give up."
