Kuvan, Naglazyme and Aldurazyme Data to Be Presented at the 57th Annual Meeting of the American Society of Huma...(sion study reported safety and eff...)

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

function.

-- 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

observed (p<0.001).

-- A sustained reduction in urinary GAGs was observed in both treatment

groups.

-- 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

(Grange)

-- 2230 -- Safety and efficacy of sapropterin dihydrochloride

(sapropterin) treatment over 22 weeks in patients with phenylketonuria

(PKU) (Randolph)

-- 2231 --
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