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Sanfilippo Syndrome: BioMarin Pharmaceutical’s FDA Orphan Drug Designation

November 27, 2014

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 B (MPS IIIB, Sanfilippo Syndrome Type B)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: BioMarin Pharmaceutical, Inc. 105 Digital Drive Novato, CA

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Please Note: “Chemicals in Flasks by Joe Sullivan (Flickr) [CC-BY-2.0] | Wikimedia Commons.

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3 Comments
  1. My daughter has MPSIIIB but it is the attenuated form. Also know of several others by word of mouth. I have forwarded this so it may help others. I am really interested in BioMarin at this time.

    • Thanks for visiting the Blog & contributing your comments. BioMarin Pharmaceutical is a great biotech to follow specifically for rare disease treatments.

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