BioMarin Pharmaceutical Inc announced 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 programme 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. "Enrolment 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 programme."

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 three consecutive 12-week dosing intervals. The objectives of the phase-1/2 study will be to evaluate safety, pharmacokinetics, pharacodynamics and to identify the optimal dose of GALNS for future studies.

BioMarin has developed and manufactures two FDA-approved enzyme replacement therapies, one for the treatment of MPS I and one for the treatment of MPS VI. Naglazyme (galsulfase) for MPS VI is wholly developed and commercialized by BioMarin. Aldurazyme (laronidase) for MPS I is manufactured by BioMarin and marketed by Genzyme Corporation.

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three approved products and multiple clinical and preclinical product candidates.