BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld Science
  • BioWorld Asia
  • Data Snapshots
    • Biopharma
    • Medical technology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • NME Digest
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Ebola outbreak
    • Hantavirus
    • Trump administration impacts
    • Med-tech outlook 2026
    • Under threat: mRNA vaccine research
    • BioWorld at 35
    • Biopharma M&A scorecard
    • Bioworld 2025 review
    • BioWorld MedTech 2025 review
    • BioWorld Science 2025 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
BioWorld - Thursday, June 11, 2026
See today's BioWorld
Home » AZ gets CRL asking for REMS changes to rare disease sBLA
To read the full article sign up for free or sign in.

AZ gets CRL asking for REMS changes to rare disease sBLA

Sep. 6, 2023
By Lee Landenberger
While the U.S. FDA didn’t ask for more study data or have safety or efficacy concerns, it does want modifications to Alexion, Astrazeneca Rare Disease’s sBLA for Ultomiris (ravulizumab-cwvz) to treat adults with the rare central nervous system disease neuromyelitis optica spectrum disorder. The agency has issued a complete response letter (CRL) requesting changes to Ultomiris’ Risk Evaluation and Mitigation Strategy (REMS) to better validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics before being treated.
BioWorld Regulatory Neurology/psychiatric Rare disease FDA

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for June 10, 2026.
  • News in brief

    BioWorld Asia
    BioWorld Asia briefs for June 9, 2026
  • 3D rendering of β2-Adrenergic receptor GPCR protein molecule embedded in lipid bilayer membrane.

    Skape Bio unlocks GPCR targets with de novo-designed miniproteins

    BioWorld
    Modulating G protein-coupled receptors (GPCRs) is one of the major challenges in biomedicine. These are flexible proteins with small, deep binding pockets. The...
  • Antares Therapeutics divulges new CDK2/cyclin E1 inhibitors

    BioWorld Science
    Antares Therapeutics Inc. has reported new CDK2/cyclin E1 inhibitors potentially useful for the treatment of cancer.
  • Empire Discovery Institute, Univ. of Rochester identify IRAK inhibitors

    BioWorld Science
    Empire Discovery Institute and the University of Rochester have discovered new interleukin-1 receptor-associated kinase 1 (IRAK-1) and/or IRAK-4 inhibitors...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Medical technology
    • Newco news
    • Opinion
    • Regulatory
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/metabolic
    • Immune
    • Infection
    • Neurology/psychiatric
    • NME Digest
    • Patents
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2026. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing