Aldurazyme

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BioMarin
Aldurazyme
Recombinant human a-L-Iduronidase
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Milestones

• Application Received July 29, 2002
• First Action Due Date January 28, 2003

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AldurazymeDrug Substance

• Recombinant human a-L-iduronidase
•   Single peptide with a molecular weight of 83
  kDa
• Produced in a continuous Chinese Hamster Ovary
  cell line transfected with cDNA encoding
  a-L-iduronidase
• Sequence is identical to a natural polymorphism
  of the endogenous protein (1 Glutamine to
  Histidine substitution)
• Six N-linked complex oligosaccharide sites
• One disulfide bond
• Well characterized and highly purified molecule

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AldurazymeDrug Product
Sterile solution intended for intravenous
administration Each vial of Drug Product
contains 2.90 mg a-L-iduronidase 43.9 mg
sodium chloride USP/EP 63.5 mg sodium phosphate,
monobasic monohydrate USP/EP 10.7 mg sodium
phosphate, dibasic heptahydrate USP/EP 0.05 mg
polysorbate 80 NF/EP 5 ml water for injection
USP/EP pH 5.2-5.9 Storage 2-8?C Expected
dating period 24 months No outstanding Drug
Product issues
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FDA Reviewers
Product Clinical Pharm Melanie Hartsough,
Ph.D. Martin Green, Ph.D Blair Fraser, Ph.D.
Howard Anderson, Ph.D. Biostatistics Cla
ra Chu, Ph.D Clinical Ilan Irony, M.D.
Facility Deborah Trout Pharm/Tox
Cynthia Kelley, M.S. Richard Mcfarland,
M.D., Ph.D. Calvin Koerner Marlene
Swider Reg. Coordinator Katherine Needleman,
M.S. Biomonitoring Bradley Glasscock, Pharm
D. Debra Bower, MT (ASCP)