Ultragenyx Pharmaceutical announced Thursday that their investigational gene therapy demonstrated promising results in a late-stage clinical trial for patients suffering from a rare inherited liver condition.
The experimental treatment, known as DTX301, showed an 18% improvement in controlling ammonia levels after 36 weeks of treatment in patients diagnosed with ornithine transcarbamylase deficiency, also called OTC deficiency, when compared to those receiving a placebo.
OTC deficiency occurs when patients lack a crucial liver enzyme responsible for eliminating ammonia from the body. Without this enzyme, dangerous levels of ammonia accumulate in the bloodstream, potentially causing mental confusion, seizures, and even coma in severe cases.
According to the pharmaceutical company, patients generally responded well to the gene therapy treatment. The most frequently reported adverse effects involved liver complications, which medical teams successfully managed using steroid medications.
