Sanfilippo Syndrome: BioMarin Pharmaceutical’s FDA Orphan Drug Designation

Orphan Druganaut Blog

BioMarin Pharmaceutical receives FDA Orphan Drug Designation (ODD) on November 25th for its investigational Enzyme Replacement Treatment (ERT), BMN-250, for the treatment of Mucopolysaccharidosis III Type B (MPS IIIB, Sanfilippo Syndrome Type B). BioMarin Pharmaceutical expects to start clinical studies with BMN-250 in mid-2015.

“BMN-250 is a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or Mucopolysaccharidosis type IIIB (MPS IIIB). 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 Pharmaceutical website)

FDA ODD Database Record For BioMarin Pharmaceutical

Generic Name: Chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2
Trade Name: n/a
Date Designated: 11-25-2014
Orphan Designation: Treatment of mucopolysaccharidosis III Type…

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