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Replagal TM Gene Activated Human a Galactosidase

Transkaryotic Therapies, Inc. Replagal TM Gene Activated Human a Galactosidase . Milestones. Application Received: June 16, 2000 1 st Complete Response (CR) Letter: December 22, 2000 TKT’s Response to 1 st CR Letter: May 24, 2002 2 nd CR Letter: November 22, 2002.

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Replagal TM Gene Activated Human a Galactosidase

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  1. Transkaryotic Therapies, Inc. ReplagalTM Gene Activated Human aGalactosidase

  2. Milestones • Application Received: June 16, 2000 • 1st Complete Response (CR) Letter: December 22, 2000 • TKT’s Response to 1st CR Letter: May 24, 2002 • 2nd CR Letter: November 22, 2002

  3. ReplagalTMDrug Substance • Gene activated Human alpha galactosidase • Homodimer comprised of two approximately 50 kDa subunits • Produced in a continuous human cell line • Identical amino acid sequence to endogenous protein • Three N-linked glycosylation sites • Well characterized molecule • No outstanding Drug Substance issues

  4. ReplagalTMDrug Product • Sterile solution intended for intravenous administration • Each vial of Drug Product contains: § 3.5 mg alpha galactosidase § 12 mg sodium phosphate USP § 0.8 mg polysorbate 20 USP § 31 mg sodium chloride USP • Isotonic • pH 5.4 – 6.6 • No outstanding Drug Product issues

  5. FDA Review Committee • ProductClinical • Blair Fraser, Ph.D. Dwaine Rieves, M.D. • John Hill, Ph.D. Marc Walton, M.D., Ph.D. • Barry Cherney, Ph.D. • Gary Kikuchi, Ph.D. Pharm/Tox • Rona LeBlanc, Ph.D. Mercedes Serabian, M.S., DABT • Ralph Bernstein, Ph.D. • Clinical Pharm • Biostatistics Martin Green, Ph.D. • Ghanshyam Gupta, Ph.D. • Biomonitoring • Facility Debra Bower, MT (ASCP) • Daniel Kearns • John Eltermann, R.Ph., M.S. Reg. Coordinators Bradley Glasscock, Pharm D. • Katherine Needleman, M.S. • Jeanne Delasko, RN, MS • Mike Harlow, B.S.

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