JACKSONVILLE, Fla. — Four-year-old Harlow Higbee’s parents are facing the unsettling prospect of losing access to a treatment they credit with significantly enhancing their daughter’s well-being. They fear that the potential unavailability of DCA could have dire consequences not only for Harlow but for other children suffering from similar conditions.

Harlow was diagnosed with an exceedingly uncommon genetic mitochondrial disorder shortly after birth. Her parents, Kim and Rick Higbee, have witnessed remarkable improvements in her health since she began taking DCA.

Kim Higbee explained, “Harlow’s condition is known as pyruvate dehydrogenase complex deficiency, or PDCD.” This disorder interferes with her ability to process carbohydrates efficiently.

“Instead of breaking down carbohydrates normally, her body converts them into lactic acid,” added Rick Higbee. The couple remains hopeful that the FDA will reconsider its stance, emphasizing the profound impact DCA has had on their daughter’s life.

Harlow was diagnosed with the life-threatening disease at just nine months old.

“Harlow, she’s four, and the statistics for PDCD — 90% could pass by their 4th birthday. We’ve already passed her 4th birthday, so every day is a gift for us with Harlow,” Kim said.

Today, Harlow’s parents say she has energy, her mind is sharper and she can now play with toys.

“I remember her first birthday, we told people don’t buy toys because she doesn’t play with toys. Now she has a room full of toys — everything is her playground,” Kim said. 

“She just said ‘mama’ for the first time last week.”

Harlow’s parents credit the investigational drug DCA, first received during a clinical trial, with giving her this chance to live more fully.

“It helps give her, in a nutshell, a boost of extra energy,” Rick said.

“It’s a lifeline to us, honestly. It’s hope,” Kim said. “100% it’s hope for any family when there’s a possible treatment for a child where there is no cure.”

But the FDA recently denied approval for DCA, saying data from the two clinical trials submitted by the drugmaker isn’t strong enough to prove effectiveness for PDCD patients. The FDA is requesting another clinical trial.

“Many drugs for rare diseases are supported by small companies that have limited funds. If this drug is not approved the next time we try, it’s unlikely that Saol Therapeutics will have the resources to continue providing DCA, which means that the provision of clinically grade DCA will stop — not only for the Higbee family,  but also for other families currently relying on Saol’s supply,” Dr. Peter Stacpoole with the University of Florida said. He led the DCA clinical trial.

“It’s heartbreak. It feels like our diagnosis all over again,” Kim said. “It’s kind of ‘where do you go next.’”

More than 80 scientific and medical leaders, including Dr. Stacpoole, signed a letter urging the FDA to act urgently on DCA approval for PDCD.

“In those children who had very severe muscle weakness, the drug was statistically significantly effective in improving muscle function,” Dr. Stacpoole said. “ We found DCA was totally safe and well-tolerated by these children. There were no serious adverse events associated with the drug. And all of the children survived.”

Due to the government shutdown, the FDA says media responses are delayed, so we have not yet gotten a response.

“What do you want lawmakers to know?” Heather Crawford asked.

“We have a safe and effective and relatively inexpensive oral agent that can improve morbidity and mortality in these at-risk children,” Dr. Stacpoole said. “There is no alternative. We either have to get an approval or this drug goes away. And that would be a tragedy.”

The Higbees are asking lawmakers for help, not just for Harlow but for all children relying on DCA.

“We’re asking Congress and legislators to just push back on the FDA, just ask them for more flexibility. What we want as parents and caregivers is to meet with the FDA — to hear from us. Clinical trials are spreadsheets, data — black and white on paper,” Kim Higbee said. “Our kids, you know, come see them in color. Let us tell you the stories of what the drug has done for them.”

The Higbees say children like Harlow need the FDA to take action quickly.

“We’re just asking for them to look at rare disease differently. We can’t go back to the drawing board. We can’t have another clinical trial. We don’t have millions of dollars to come up with a whole another trial. We don’t have time. Our kids don’t have time, unfortunately.”

According to government statistics, only about 5% of nearly 10,000 identified rare diseases have FDA-approved treatments.

“As any parent knows, when there is something wrong with your child, you would move heaven and earth to get answers and provide them with the care they need,” U.S. Congressman John Rutherford said. “My office has been in contact with the Higbee family and Soal Therapeutics about the FDA’s complete response letter for dichloroacetate to treat PDCD. I look forward to working with them to promote compassion and regulatory flexibility at the FDA.”

Saol Therapeutics says it does not plan to conduct another clinical trial at this time. It plans to file a request with the FDA for a meeting, during which the company says it will present “strong data” the FDA has not seen yet. In the meantime, patients’ access to DCA will continue through the ongoing open-label extension and the expanded access program.

Saol Therapeutics’ Full Statement

“Saol Therapeutics has received a Complete Response Letter (CRL) from the FDA for its NDA for SL1009 (sodium dichloroacetate, or DCA). We are working toward a meeting with the Agency in the coming weeks to address the feedback outlined in that letter, which primarily centers on the quantity and type of clinical evidence available for such a small patient population. The existing data supporting SL1009 is very strong, reflecting more than four years of clinical experience across two Phase III studies, an ongoing open-label extension, and survival analyses compared to natural history. In preparation for our meeting, we are compiling additional analyses of this data, and we do not plan to conduct another clinical trial at this time. As we work through next steps with the FDA, patient access to SL1009 will continue through both the ongoing open-label extension and the Expanded Access Program.”