UCB Drug Becomes First FDA-Approved Treatment for Ultra-Rare and Fatal Mitochondrial Disease

The energy that powers cells of the human body comes from tiny cellular components called mitochondria. Just as a draining battery makes it difficult to operate an electronic device, mitochondrial disorders impair cellular function and cause progressively worsening muscle weakness that diminishes a patient’s ability to move, swallow, and even breathe. One particular mitochondrial depletion syndrome now has its first FDA-approved therapy , a drug acquired by UCB as part of a $1.9 billion deal.

UCB’s Kygevvi is approved for treating thymidine kinase 2 deficiency (TK2d) in adults and children whose disease symptoms started at age 12 or earlier. In addition to becoming the first approved therapy for TK2d, Kygevvi is also the first ultra-rare disease drug for Brussels, Belgium-based UCB.