Ultragenyx Pharmaceutical Inc. Wins 2nd Drug Approval in 2 Weeks

Ultragenyx Pharmaceutical Inc. won its second drug approval in two weeks — and a month ahead of schedule — as regulators greenlighted a treatment against a family of debilitating and potentially deadly genetic disease.

The Novato-based company (NASDAQ: RARE) said a 500-milliliter bottle of the drug to treat long-chain fatty acid oxidation disorders will cost $4,875 wholesale, or an average net price of $138,000 per patient per year.

Because the disorders can occur in newborns, Ultragenyx said the drug, branded as Dojolvi, will have an average net price of $46,000 in the first year of a patient’s life. A company spokesman said the “vast majority” of patients will have zero or “very low” out-of-pocket costs for the drug.

Ultragenyx’s stock closed up $4.56 per share, or 6.2%, to $78.22.

The FDA wasn’t expected to make a decision on the drug until July 31.

Long-chain fatty acid oxidation disorders are a group of rare conditions that prevent the body from breaking down certain fats into energy, leaving kids with low blood sugar, muscle ruptures and muscle weakness and heart disease. They are estimated to affect one in 9,300 people in the United States, Australia and Germany, with some 3,000 to 3,500 children and adults in the U.S. alone.

Ultragenyx earlier this month won FDA approval to sell its already-approved drug Crysvita to treat tumor-induced osteomalacia, a rare disease that causes weakened or soft bones.