{"id":60562,"date":"2025-07-12T21:26:16","date_gmt":"2025-07-12T21:26:16","guid":{"rendered":"https:\/\/www.europesays.com\/us\/60562\/"},"modified":"2025-07-12T21:26:16","modified_gmt":"2025-07-12T21:26:16","slug":"georgia-lawmakers-unite-to-urge-fda-reversal-on-drug-denial-for-child-with-rare-disease","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/us\/60562\/","title":{"rendered":"Georgia lawmakers unite to urge FDA reversal on drug denial for child with rare disease"},"content":{"rendered":"

\"Family Family fights for experimental drug for 4-year-old girl<\/a> <\/p>\n

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.<\/p>\n

GAINESVILLE, Ga.<\/strong> – Georgia\u2019s 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<\/a>.<\/p>\n

What they’re saying:<\/p>\n

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.<\/p>\n

“Basically, we started emailing every single member of our delegation,” said Haley Bower, Hope\u2019s aunt.<\/p>\n

That determination led to a bipartisan letter from the state\u2019s congressional delegation, addressed to the FDA, questioning the agency\u2019s rejection of elamipretide and the process by which it made its decision.<\/p>\n

Rep. Buddy Carter, a pharmacist by training and chair of the Health Subcommittee on Energy and Commerce\u2014which has oversight of the FDA\u2014said he understands the complexities involved.<\/p>\n

“Being a pharmacist, I understand the process of how drugs are approved by the FDA,” Carter said.<\/p>\n

What we know:<\/p>\n

One major challenge is the rarity of Hope\u2019s 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.<\/p>\n

“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\u2019s Hospital of Philadelphia.<\/p>\n

Carter said patients with rare diseases deserve careful consideration, even when potential treatments won\u2019t benefit millions.<\/p>\n

“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.”<\/p>\n

Hope\u2019s family says they\u2019re encouraged by the bipartisan support.<\/p>\n

“This has nothing to do with political motives or ideology,” said her father, Ben Filchak. “It\u2019s just the right thing to do.”<\/p>\n

Filchak praised Rep. Lucy McBath, a Democrat, for being a strong advocate for the rare disease community.<\/p>\n

“We had a great conversation with Congresswoman McBath,” he said. “She is a huge advocate for the rare disease community.”<\/p>\n

What we don’t know:<\/p>\n

While the outcome remains uncertain, the family remains cautiously optimistic.<\/p>\n

“We\u2019re still nervous, you could say, about the outcome,” Filchak said. “But I absolutely think this will help.”<\/p>\n

The Source:<\/b> FOX 5’s Kevyn Stewart spoke with the family of Hope Filchak and Rep. Buddy Carter. Previous FOX 5 Atlanta reporting was also used.<\/p>\n

Health<\/a>Health Care<\/a>News<\/a>Hall County<\/a> <\/p>\n","protected":false},"excerpt":{"rendered":"Family fights for experimental drug for 4-year-old girl A 4-year-old girl from Hall County is getting some powerful…\n","protected":false},"author":3,"featured_media":60563,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[34],"tags":[17505,13105,210,1141,1060,50,67,132,68],"class_list":{"0":"post-60562","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-medication","8":"tag-ga","9":"tag-hall-county","10":"tag-health","11":"tag-health-care","12":"tag-medication","13":"tag-news","14":"tag-united-states","15":"tag-unitedstates","16":"tag-us"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@us\/114842382275039557","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/posts\/60562","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/comments?post=60562"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/posts\/60562\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/media\/60563"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/media?parent=60562"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/categories?post=60562"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/tags?post=60562"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}