Liprotamase: Phase III data

In 145 CF patients who completed 12 months of treatment with liprotamase in an open-label, international Phase III trial, 96% of patients maintained or gained weight at the same rate as the healthy U.S. population. In patients 7-20 years of age, 99% maintained or gained weight. Liprotamase also maintained nutritional status as measured by body mass index (BMI) and showed no significant differences from baseline in levels of micronutrients such as albumin, prealbumin, triglycerides, cholesterol, alpha-tocopherol and vitamins A, D and K at 6 and 12 months. The company said these data would support efficacy data from a Phase III trial that met the primary endpoint of a significant improvement in fat absorption as measured by the coefficient of fat absorption (CFA) (see BioCentury, Aug. 18, 2008).

Patients took an average of 5.4 capsules/day; the company said CF patients take about 20 capsules/day of the current porcine-derived pancreatic enzyme replacement therapies. The trial enrolled 215 patients, 69 of whom withdrew early. There were 37 withdrawals due to adverse events, which the company said were expected and primarily gastrointestinal and pulmonary exacerbations. There was 1 undisclosed gastrointestinal treatment-related serious adverse event that self-resolved. ...