Try our mobile app
SAN RAFAEL, Calif., Dec. 21, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that children in the open-label long-term extension of the Phase 3 study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP), maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment. These analyses are the result of the combination of data of the same patients enrolled in three consecutive trials. In the first trial, a "run in" period consisted of longitudinal measurement of height in all patients prior to receiving treatment. After at least six months observation in the run-in trial, 121 patients were randomized 1:1 to receive either placebo or vosoritide at a dose of 15 ug/kg/day. One year later, patients previously receiving placebo were crossed over to receive vosoritide in an open-label treatment extension study, while those patients previously on vosoritide remained on treatment.
A first analysis, comparing all children randomized and treated with vosoritide for two years (n=52) to all children from the run-in study who were randomized to receive placebo with an untreated observation period of two years (n=38), showed improvement in one-year height change in the treated group relative to the untreated group that was similar in the second year of treatment, 1.79 cm, as in the first year of treatment, 1.73 cm. The cumulative height gain over the 2-year treatment period was 3.52 cm compared to untreated children, which is the sum of the first year (1.73) and the second year (1.79).
Yearly change in standing height (cm) | Year 1 | Year 2 |
Untreated (N=38*), mean (SD) | 3.96 (0.92) | 3.82 (0.99) |
Vosoritide (N=52**), mean (SD) | 5.69 (0.97) | 5.61 (1.09) |
Treatment effect (95% CI) | 1.73 (1.33 - 2.14) | 1.79 (1.35 - 2.24) |
P-value*** |
|
