Family fights for experimental drug for 4-year-old girl
A 4-year-old girl from Hall County is getting some powerful help in her fight to survive. Last month, we told you about Hope Filchak. She’s living with a very rare disease. The family is fighting to get an experimental drug approved by the FDA, but it has already been rejected once.
GAINESVILLE, Ga. – Georgia’s congressional delegation is joining forces across party lines in an effort to help a 4-year-old Hall County girl battling a rare and life-threatening illness.
What they’re saying:
Hope Filchak was born with a mitochondrial disorder known as MLS, which has left her blind, deaf, and with two serious heart defects. Her family says an experimental drug called elamipretide has helped her, but the U.S. Food and Drug Administration has already rejected its approval once.
“Basically, we started emailing every single member of our delegation,” said Haley Bower, Hope’s aunt.
That determination led to a bipartisan letter from the state’s congressional delegation, addressed to the FDA, questioning the agency’s rejection of elamipretide and the process by which it made its decision.
Rep. Buddy Carter, a pharmacist by training and chair of the Health Subcommittee on Energy and Commerce—which has oversight of the FDA—said he understands the complexities involved.
“Being a pharmacist, I understand the process of how drugs are approved by the FDA,” Carter said.
What we know:
One major challenge is the rarity of Hope’s illness. Because so few patients exist, it’s difficult to conduct large-scale clinical trials that meet FDA benchmarks. One such benchmark, the six-minute walk test, failed to show statistically significant improvement in trials.
“The one the FDA cares about the most is the six-minute walk test that did not meet statistical significance,” said Dr. Amy Goldstein of Children’s Hospital of Philadelphia.
Carter said patients with rare diseases deserve careful consideration, even when potential treatments won’t benefit millions.
“They deserve as much attention as anyone,” he said. “Even if this were a blockbuster drug that was going to help millions of people, we still need to be looking at this carefully.”
Hope’s family says they’re encouraged by the bipartisan support.
“This has nothing to do with political motives or ideology,” said her father, Ben Filchak. “It’s just the right thing to do.”
Filchak praised Rep. Lucy McBath, a Democrat, for being a strong advocate for the rare disease community.
“We had a great conversation with Congresswoman McBath,” he said. “She is a huge advocate for the rare disease community.”
What we don’t know:
While the outcome remains uncertain, the family remains cautiously optimistic.
“We’re still nervous, you could say, about the outcome,” Filchak said. “But I absolutely think this will help.”
The Source: FOX 5’s Kevyn Stewart spoke with the family of Hope Filchak and Rep. Buddy Carter. Previous FOX 5 Atlanta reporting was also used.
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