Fall25BowerFamily

Georgia Family Urges Action After FDA Denies Life-Saving Rare Disease Drug

Georgia Association of Convenience Stores (GACS) member Haley Bower of Clipper Petroleum, Inc. is leading a fight to save a drug that has transformed the life of her 4-year-old niece, Hope Filchak. Hope lives with Microphthalmia with Linear Skin Defects (MLS) syndrome, an extremely rare mitochondrial disease.

Hope’s condition has caused deafness, blindness, agenesis of the corpus callosum, Wolff-Parkinson-White syndrome, and cardiomyopathy. For the first three years of her life, her heart was stable, but in January 2024, her ejection fraction dropped from 60% to 46%, signaling the progression of heart failure. Her medical team at the Children’s Hospital of Philadelphia (CHOP) immediately approved her for elamipretide, an investigational drug available under the FDA’s Expanded Access Program.

The change was dramatic. Before treatment, Hope often slept 17 hours a day, missed school, and fell behind in therapy. Now, she’s alert, thriving, and attending school daily. Her heart function has stabilized, her BNP levels have dropped, and she’s participating in activities like music class and gym for the first time.

But Hope’s future is uncertain. Despite a 10–6 FDA advisory committee vote in October 2024 recommending full approval for elamipretide in Barth syndrome, a related rare disease, the FDA issued a Complete Response Letter on May 29, 2025, denying approval. Advocates say this decision threatens access for patients already benefiting from the drug and sets a dangerous precedent for rare disease innovation. StealthBioTherapeutics, a clinical-stage biotechnology company focused on therapies for mitochondrial diseases and the developer of elamipretide, resubmitted its New Drug Application (NDA) in mid-August. The FDA accepted the filing the following week and announced it would issue a decision on the drug’s approval by September 26, just weeks away.

“This isn’t just about Hope,” Haley said. “This is about every child and family living with a rare, life-threatening disease. Without elamipretide, these children face decline and death.”

Hope’s family has appealed to Senator Jon Ossoff, HHS Secretary Robert F. Kennedy Jr., and FDA Commissioner Dr. Marty Makary, urging congressional oversight and action to restore access and trust in the FDA’s review process.

With strong support from the community, Hope’s family worked tirelessly and successfully secured a bipartisan letter. They personally met with all 14 congressional offices to ensure their voices were heard and to bring attention to Haley’s story. In addition, Governor Brian Kemp, Lt. Governor Burt Jones, and Speaker Jon Burns sent a joint letter to Commissioner Makary and Secretary Kennedy, reinforcing the call for fairness, transparency, and compassion in the FDA’s decision-making process (click here to view letter).

But the fight does not stop at securing approval for elamipretide. Advocates are also pushing for more inclusive drug labeling. Under current guidelines, strict age restrictions would prevent Hope from using elamipretide even if it were approved, leaving her and children like her vulnerable. Advocates say labeling reform is critical to ensuring lifesaving treatments are accessible to the youngest and most fragile patients.

“Hope is a miracle,” Haley said. “We’re asking for fairness, compassion, and the chance for her, and children like her, to live a full life.”

For now, Hope is able to receive elamipretide through the FDA’s Expanded Access Program, but that safety net is fragile. Without urgent action from regulators and lawmakers, the very medication giving her stability and a childhood could be stripped away, leaving families like hers out of options.

The Filchak family, along with advocates nationwide, is calling on lawmakers, regulators, and the public to demand transparency from the FDA and prioritize treatments for ultra-rare diseases. For families like theirs, this isn’t about policy—it’s about survival. Every day without action puts children like Hope at risk, and her family says they won’t stop fighting until the drug that gave her life is secured for her future.