RICHLAND, Miss. (WLBT) – A Rankin County family’s fight to get a drug approved takes them to Washington, D.C., where they had a sit-down with the FDA Commissioner.
After a peaceful demonstration with other families impacted by Barth Syndrome, they have hope for future access to a medication denied by the agency.
“We were there on a wing and a prayer, and we got it,” said Kristi Pena.
The 44-year-old and her sister, Amy Wilson, both having sons with Barth Syndrome, traveled to the nation’s capital to the Food and Drug Administration to approve the drug Elamipretide.
They hoped to meet FDA Commissioner Dr. Martin Makary, but it was a long shot.
“Out of 75 letters written requesting a face-to-face meeting, my letter was chosen for a face-to-face,” said Pena.
They were joined by five other Barth Syndrome families and the organization’s executive director, sharing their experiences and Elamipretide data with Makary.
“It was an empowering experience,” said Pena. “We felt out of nine years of this battle with the FDA, finally we felt heard. We feel hopeful that there will be an answer.”
The women learned that the FDA will decide on the drug by September 26.
Elamipretide, which was previously discontinued, will now be available through the end of September.
In August, the manufacturer, Stealth Bio Therapeutics, announced that their doors would be closed and there would be no access to the medication after the end of August.
“Once we got to D.C., we had a meeting with the Barth Syndrome Foundation that told us that the drug manufacturer had indeed filed another drug application, [and] that they had 30 days to accept that application. They [the FDA] did so in less than a week, which was very encouraging for us,” said Amy Wilson.
After six days in D.C., they are optimistic the life-changing drug will get approved this third time, but said it needs to be extended to children two to 12 years old.
“Hopefully, a good resolution because we’re not gonna stop until this medication is approved for all ages,” added Pena.
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