BioMarin commences GALNS Phase 2 study in mucopolysaccharidosis IVA

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BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the initiation of a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA (MPS IVA). 

"We expect this study to build our knowledge base in enzyme replacement therapies wherein clinicians believe earlier therapy is more impactful," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin.  "Comparatively speaking, this study is starting years before a similar study for Naglazyme, which was done as a post-marketing commitment.  We will also conduct a study in non-ambulatory patients, which, along with the study in patients under five years of age, will encompass the spectrum of MPS IVA patients not included in the Phase 3 trial.  Enrollment in the Phase 3 pivotal study is on track for results in the second half of 2012, and its execution is BioMarin's highest product development priority."

The primary objective of the Phase 2, open-label, multinational clinical study is to evaluate the safety and tolerability of infusions of GALNS at a dose of 2.0 mg/kg/week over a 52-week period in ten to fifteen MPS IVA patients under five years of age.  The secondary objectives are to evaluate urinary keratan sulfate (KS) levels and growth velocity.

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