June 30 Quick Takes: Another first for Ultragenyx; plus approvals for Genentech’s Phesgo, Keytruda, Bavencio and Ultomiris

Ultragenyx’s Dojolvi wins FDA approval for rare disease

FDA approved Dojolvi triheptanoin (UX007) from Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) to treat long-chain fatty acid oxidation disorders, making it the first drug approved for the indication. The therapy is a synthetic compound that provides medium-length, odd-chain fatty acids that are metabolized to replace intermediate substrates in fatty acid oxidation and in the tricarboxylic acid cycle. Ultragenyx said it expects the drug to be available next month at a wholesale acquisition cost per bottle of $4,875, or an average net price of $138,000 per year before discounts and dosing adjustments based on caloric intake that could reduce cost to patients. It is the company’s fourth FDA approval in the past three years, all of which have been for rare diseases with no approved therapies. ...