efficacy data of the 24-week pivotal Phase 3 trial, followed by a
72-week extension study for a total of 96 weeks.
-- Endpoints included 12-minute-walk-test (12MWT), 3-minute-stair-climb
(3MSC), level of urinary glycosaminoglycans (GAGS) and pulmonary
-- Patients receiving Naglazyme improved by a mean of 183 m +/- 26 m from
baseline to week 96 in the 12MWT (p<0.001).
-- The placebo group, which was switched to active drug at week 24,
improved by a mean of 117 m +/- 25 m from week 24 to week 96 (p<0.001).
-- Similar improvements in the rate of stairs climbed (3MSC) were also
-- A sustained reduction in urinary GAGs was observed in both treatment
-- Several pulmonary function measures showed improvement at Week 96,
including forced vital capacity (FVC), which improved 0.11 L +/- 0.05 L
(p=0.039) in the Naglazyme group from baseline and improved 0.07 L +/-
0.02 L (p<0.001) in the placebo/Naglazyme group from Week 24.
-- Safety data for the extension study was similar to that measured in the
first 24 weeks of treatment. The overall safety profile remained
favorable. Adverse events reported by the greatest number of patients
during the extension study included headache, cough, pyrexia, diarrhea,
arthralgia and extremity pain. None of the adverse events that
occurred during infusion were severe.
The following relevant posters will also be featured:
-- 2229 -- Safety of sapropterin dihydrochloride (sapropterin) in children
with phenylketonuria (PKU) on a phenylalanine (Phe)-restricted diet
-- 2230 -- Safety and efficacy of sapropterin dihydrochloride
(sapropterin) treatment over 22 weeks in patients with phenylketonuria
-- 2231 --
|SOURCE BioMarin Pharmaceutical Inc.|
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