Struggle of a Colorado family for obtaining a life-saving medication for their infant afflicted with Barth syndrome
In a heartwarming yet urgent tale, the Dryden family from Colorado is waiting anxiously for a decision from the Food and Drug Administration (FDA) on elamipretide, an experimental drug that has shown significant promise in improving the health of individuals with Barth syndrome, a rare genetic condition that primarily affects boys and can lead to heart failure, even in infancy.
Gilbert Dryden, a young infant born with Barth syndrome, has been receiving a three-month supply of elamipretide, manufactured by Stealth BioTherapeutics Inc. The family is concerned about running out by the end of October, as the FDA has yet to make a final decision on the drug's approval.
The journey for elamipretide began in 2015, when Dr. Brian Stauffer, a cardiologist and researcher at the University of Colorado Anschutz Medical Campus, collaborated with Dr. Kathryn Chatfield and others on initial research into the drug. Dr. Chatfield, a pediatric cardiologist at Children's Hospital Colorado, was instrumental in identifying Gilbert's condition.
The family's hopes were raised when, within a month of being on elamipretide, Gilbert showed immediate and dramatic improvements in his health. He was able to get his NG tube out and come home, a significant milestone for the family.
The FDA agreed to reconsider elamipretide's application under an accelerated pathway, following a recommendation from an advisory panel last October. However, the FDA delayed final approval, citing the small trial size and requesting more data.
Rep. Jason Crow is leading a bipartisan push for action, urging the FDA to make a timely decision on elamipretide. All of Colorado's congressional delegation have signed on to a letter urging the FDA to expedite the process.
The delay has raised concerns from the medical community. Dr. Chatfield expressed her worry, stating, "We do not have the luxury of waiting many more months or years." The FDA did not respond to a request for comment when contacted for this story.
The story of Gilbert and his family resonates deeply with Madison Dryden, who lost her brother Ben to heart failure as a baby due to an unknown condition. When Gilbert was admitted to the NICU shortly after birth, Madison's thoughts went immediately to her late brother Ben.
As the decision date for elamipretide approaches on Sept. 26, the Dryden family, along with their supporters, hold onto hope for a positive outcome. They are not alone in their quest, as the future of this potentially life-saving drug hangs in the balance.
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