Ultragenyx scores 2nd FDA approval in as many weeks for ultra-rare metabolic disease drug Dojolvi

Rare disease drugmaker Ultragenyx scored a big win just days ago with Crysvita's FDA approval in an ultra-rare bone disorder. Now, the company has struck gold again, this time with a new drug targeting a tiny group of metabolic disorders. 

The FDA on Tuesday approved Ultragenyx's Dojolvi (triheptanoin) as a metabolic supplement for patients with a rare group of disorders who cannot convert certain fatty acids into energy, the company said. 

Dojolvi, a synthetic medium-chain triglyceride, acts as an energy source for patients who cannot metabolize long-chain fatty acids, including beneficial omega-3's, on their own. The resulting extreme depletion of glucose in the body can lead to hospitalizations and even early death, Ultragenyx said. 

The group of disorders is believed to affect between 2,000 and 3,500 U.S. children each year, the drugmaker said. 

According to Ultragenyx CEO Emil Kakkis, Dojolvi's unique mechanism of action will be a game-changer for patients with the family of ultra-rare disorders who have typically relied on restrictive diets to reduce their symptoms. 

"It's a smart way to engineer a drug to ... bypass the defect to create energy and function," Kakkis said. 

RELATED: Ultragenyx's Crysvita scores FDA nod in 2nd ultra-rare bone disorder