BioMarin's Clinical Trial Application for GALNS for Morquio A Syndrome Accepted b...( NOVATO Calif. March 18 /- BioMarin Ph...)

NOVATO, Calif., March 18 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that its application for clinical trial authorization (CTA) for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome, has been accepted by the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA). BioMarin expects to initiate a Phase 1/2 clinical trial in the next few weeks.

"There are a significant number of untreated Morquio patients in clinics around the world who are very anxious to receive a new treatment option. We are moving forward expeditiously with our GALNS program in the hope of bringing those patients the much-needed treatment they deserve. BioMarin has successfully advanced two enzyme replacement therapies from IND filing to FDA approval in approximately five years each and we plan to leverage our clinical, manufacturing and regulatory expertise to develop this treatment for Morquio A syndrome," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Enrollment in the MorCAP study initiated last November is continuing and we continue to add additional sites. We expect that this survey study will yield valuable information on Morquio patients and will aid in our scientific understanding of the disorder as we further pursue this program."

The Phase 1/2 study is designed as an open-label, within-patient dose escalation trial in approximately 20 patients followed by a treatment continuation phase. During the dose escalation phase of the study, subjects will receive weekly intravenous infusions of BMN 110 in 3 consecutive 12-week dosing intervals. The objectives of the Phase 1/2 study will be to evaluate safety, pharmacokinetics, pharacodynamics and to
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