FDA Grants BioMarin Orphan Drug Designation for NAGLU Fusion Protein, BMN 250, for the Treatment of MPS IIIB

BioMarin Pharmaceutical Inc. BMRN announced today that the Food and Drug Administration (FDA) has granted orphan drug designation for BMN 250, a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo Syndrome Type B or Mucopolysaccharidosis IIIB (MPS IIIB). BioMarin expects to initiate clinical studies with BMN 250 in mid-2015. Discovered by BioMarin, BMN 250 is an enzyme replacement therapy using recombinant human NAGLU with an IGF2, or Glycosylation Independent Lysosomal Targeting (GILT) tag. BMRN 250 is delivered directly to the brain using BioMarin's patented technology. "BioMarin pioneered a proprietary approach to deliver large proteins directly to the brain, which bypasses the blood brain barrier and has typically proved difficult. This BioMarin technology to be used for the treatment of Sanfilippo patients builds on the experience we have gained using this approach to treat CLN2 disorder, a form of Batten disease," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "We look forward to meeting with the FDA to determine the regulatory path for BMN 250 for MPS IIIB or Sanfilippo Type B and applying our experience in developing three first-in-class therapies for different varieties of MPS diseases." "We are pleased that BioMarin is focusing on developing a therapy for Sanfilippo B patients," said Kathleen Buckley, President of the Team Sanfilippo Foundation. "Children affected by MPS IIIB or Sanfilippo B have no approved drug treatment options, and the families affected by this terminal disease are hopeful that BioMarin will quickly advance its experimental therapy, which has the possibility of making a difference." "BioMarin has been committed to developing therapies for many MPS disorders. We are encouraged that this biotechnology company is now turning its focus to MPS IIIB or Sanfilippo B Syndrome," said Barbara Wedehase, MSW, CGC, Executive Director of the National MPS Society. "BioMarin has expertise in enzyme replacement therapies and MPS disorders, and we appreciate their continued efforts to deliver a therapy for this unmet medical need."