FDA backs second rare disease drug from Ultragenyx in the space of a fortnight

The FDA has approved a second rare disease drug from California-based Ultragenyx in the space of  a fortnight, for treatment of long-chain fatty acid oxidation disorders. 

Ultragenyx said the regulator approved Dojolvi (tripetanoin) as source of calories and fatty acids for children and adults with confirmed disorders. 

This group of rare disorders, known as LC-FAOD for short, are lifelong and life-threatening, meaning that the body is unable to convert long-chain fatty acids into energy, 

Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride specifically designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD. 

LC-FAOD are included in newborn screening panels across the US and in certain European countries due to the risk for serious outcomes including death early in life. 

Other current treatment options for LC-FAOD include avoidance of fasting, low-fat/high-carbohydrate diets, carnitine and even-carbon medium-chain triglyceride (MCT) oil, a medical food product.  

The disorders affect an estimated 2,000 to 3,500 children and adults in the US. 

Ultragenyx chief medical officer Camille Bedrosian said: “With today’s FDA approval, patients living with this serious, unpredictable, and often catastrophic disease now have an approved therapy. Many patients with long-chain fatty acid oxidation disorders have difficult lives with frequent hospitalisations and major medical events despite the best current care. Now these patients have an approved treatment as an option to help manage their disease.” 

There was no word from Ultragenyx about price, although if this month’s earlier approval is anything to go by it is likely to be high. 

Ultragenyx has set a price of $160,000 per year for children and $200,000 for adults after rebates and discounts for Crysvita in tumour-induced osteomalacia, a rare type of skeletal tumour associated with chronically low phosphate levels. 

The FDA cleared Crysvita, developed with partner Kyowa Kirin, in mid-June. The drug was first approved in 2018 to treat X-linked hypophosphatemia in patients aged one year or older.