In the News ABC 7 New York: “Families of children with rare, life-threatening disease push FDA to review drug application

Thank you, investigative reporter Kristin Thorne at ABC 7 New York, for the in-depth coverage of our efforts. Please read, watch, and share this article with your networks to keep our momentum going!

Key Points from the article:

  • Three years ago, when Stealth submitted a new drug application to the FDA, the FDA refused even to review the application telling the company it didn’t have enough patients in its trials. Stealth was only able to locate 12 patients. “They want to see more patients,” Stealth BioTherapeutics CEO Reenie McCarthy said in an interview with Eyewitness News investigative reporter Kristin Thorne. “So, it’s been a bit of an impossible clinical development challenge to solve for us.”
  • McCarthy said the FDA is accustomed to reviewing new drug applications with trials with thousands of patients, which don’t exist for Barth Syndrome. Eyewitness News asked the FDA how many patients the agency would have to see in the elamipretide trials in order for the drug application to be eligible for review.
  • The FDA issued a statement not answering the question, but stating, “The FDA generally cannot confirm or deny the existence of a pending product application or discuss the status of a pending application.”
  • “For many Barth syndrome families, elamipretide represents their only hope for an affected child to have an increased chance to live into adulthood and to enjoy a life with reduced debilitating weakness and fatigue that precludes them from going to school, playing with friends, or holding a job,” Kate McCurdy, the co-founder of the Barth Syndrome Foundation, said.

The Barth community needs your support! Help us ensure the FDA hears these families’ stories and prioritizes this life-saving medication.

  • Share this blog post widely to spread awareness about Barth Syndrome.
  • Contact the FDA and your representatives in Congress – urge them to put pressure on the FDA.

Together, we can make sure no potential treatment for rare diseases gets left behind.