Orland Park Family Advocates for FDA Approval of Drug to Treat Barth Syndrome, a Rare Genetic Disease

A baby boy in the Chicago vicinity is contending with a rare medical condition and urgently requires a specific drug treatment to improve his quality of life. This medication, however, hasn’t gained FDA approval and might soon be unavailable if not approved.

The situation hinges on whether the U.S. Food and Drug Administration will give the green light to this drug, which is intended to treat Barth syndrome, a condition so rare that its approval has been delayed because of insufficient sample sizes.

The pharmaceutical company that produces the drug has said it could go out of business. For one family in Orland Park, that could spell disaster.

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Adel Mohammed, in his short life, has faced numerous challenges. Barely over a month old, he was taken to the hospital in severe respiratory distress.

“We were just observing as they attempted to intubate him,” said his mother, Nour Shaban. “During the process, he suffered cardiac arrest. It was a complete shock to us.”

Diagnosed with Barth syndrome, Mohammed is facing a rare genetic disorder predominantly affecting males, with only around 150 cases identified across the country.

While there are no currently FDA-approved treatments for Barth syndrome, a drug named Elamipretide has received orphan drug status. Mohammed has been receiving daily injections of this medication for several months.

“Even his pediatrician says he’s a miracle baby,” Shaban said. “He’s able to sit up on his own. Lift his head up on his own. He’s showing things he should be delayed with.”

According to the Barth Syndrome Foundation, without treatment, sufferers will exhibit muscle weakness, heart failure and delayed growth. Most early deaths happen in infancy.

Unfortunately, Elamipretide’s status remains in limbo as the approval process has dragged on for years now.

“We don’t know how long he might live if he doesn’t have access to this medication,” Shaban said. “Like I’ll show you his echos. He went from 10% back in March to 55% currently. He’s close to normal. This drug does work. It does save lives.”

While a spokesperson for Stealth BioTherapeutics admitted Wednesday the company’s future is at risk if the FDA doesn’t act soon, they also said they’re committed to keeping their current patients on the drug, for free, for as long as they can.

Stealth BioTherapeutics did resubmit its new drug application to the FDA this week. The FDA indicated they may not have a decision for up to six months.