The open-label, multinational, Phase 2 clinical study is designed to assess the safety and tolerability of infusions of GALNS at a dose of 2.0 mg/kg/week over a 52-week period in MPS IVA patients under five years of age.
The secondary objective of the study is determining urinary keratan sulfate (KS) levels and growth velocity.
BioMarin chief medical officer Hank Fuchs said the study would help them gain further insights into enzyme replacement therapies.
"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," Fuchs added.