FDA U-turn brings Immedica’s therapy to patients with ultra-rare disease

FDA executed another orphan drug approval U-turn this week, but this time the reversal led to an approval and a priority review voucher. On Monday, Swedish biotech Immedica said FDA had granted accelerated approval to Loargys to treat hyperargininemia in patients 2 years and up with arginase 1 deficiency.

The approval comes after a regulatory ride that took the ARG1-D product, and patients, on very different paths in the U.S. and Europe...