FDA Awaits Data On Clinical Significance Of <em>Vimizim</em> Neutralizing Antibodies

FDA Awaits Data On Clinical Significance Of Vimizim Neutralizing Antibodies

Vimizim post-marketing requirements could help FDA better understand the clinical significance of anti-drug antibodies commonly seen with enzyme replacement therapies for lysosomal storage diseases and the risks of tolerance induction therapy to manage immunogenicity.

Widespread development of anti-drug antibodies in studies of BioMarin Pharmaceutical Inc.’s Vimizim sparked debate about the role that tolerance induction therapy should play when FDA is making risk-benefit calculations about products where immunogenicity is a significant factor, FDA review documents show.

The effect of neutralizing antibodies on the long-term efficacy and safety of enzyme replacement therapies is a challenging issue for...