Decision: Favourable

Study Title:

Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitrypsin Deficiency Who Have the PiZZ Genotype, Over 48 Weeks

  • NREC Code:

    23-NREC-CT-002

  • Decision:

    Favourable

  • Meeting Date:

    25/01/2023

  • Study Type:

    CT application

  • Principal Investigator:

    Prof Noel Gerard McElvaney

  • PI Institution:

    Beaumont Hospital

  • Sponsor:

    Vertex Pharmaceuticals Incorporated

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