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[Free Online Symposium] Success Stories and Lessons Learned from Recent FDA Approvals: VOXZOGO (vosoritide) by BioMarin Pharmaceutical Inc.

Speakers: Heather Wenzel, Anu Cherukuri, Yulan Qi, Michelle Fong
Organizers: PBSS and California Life Sciences (CLS)
Date: 2022-07-27
Time: 10:00-13:00 Pacific Time
Registration fee: 0
Location: Online via Zoom
Major Sponsor:
Vendor show vendors registered to date:
Registration deadline:2022-07-27  (it will close sooner if the seating cap is reached)

About the Topic

VOXZOGO is a recombinant C-type natriuretic peptide (CNP) analog that has been modified to resist neutral endopeptidase degradation and enhance half-life. In 2021, VOXZOGO was approved for treatment of patients 2 years of age and older with Achondroplasia and open growth plates in the EU and under accelerated approval by US FDA to increase linear growth in patients 5 years and older with Achondroplasia whose growth plates are still open.

Achondroplasia is an inherited autosomal dominant condition caused by a gain-of-function mutation in the fibroblast growth factor receptor-3 gene (FGFR3), a negative regulator of endochondral bone formation. FGFR3 signaling via the MAPK pathway leads to decreased chondrocyte hypertrophy and matrix deposition and subsequently reduced skeletal growth at secondary ossification centers. VOXZOGO, similar to endogenous CNP, exerts its effect downstream of binding with NPR-B via PKGII, antagonizing the MAPK pathway at the level of Raf.

This presentation will discuss challenges and lessons learned during the development, marketing application review and approval process from the perspective of several project team members:

  • Interpreting Nonclinical Data for the Regulator (Heather Wenzel, MS, DABT, Associate Director, Pharmacology & Toxicology)

  • Clinical Pharmacology (Yulan Qi, PhD, Director, Clinical Pharmacology)

  • Value of the Integrated Summary of Immunogenicity or ISI (Anu Cherukuri, PhD, Senior

    Scientist, Clinical Immunology)

  • [Regulatory Lessons Learned] (Michelle Fong, Executive Director, Regulatory Affairs)

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