Cystic fibrosis digest - 2021

Cystic fibrosis digest - 2021

Updates from Vertex Pharmaceuticals

Trikafta (Elexacaftor/tezacaftor/ivacaftor and ivacaftor), Kaftrio (ivacaftor/tezacaftor/elexacaftor), Symkevi (tezacaftor/ivacaftor) – Vertex Pharmaceuticals

  • FDA grants expanded use approval for Trikafta based on in-vitro data
    • Approval to include children with cystic fibrosis (CF) aged 6 through 11 years who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive to Trikafta (For full story click here)
  • Health Canada granted marketing authorization for Trikafta in people ages 12 and older with at least one F508del mutation (For full story click here)
  • Vertex announced a national reimbursement agreement in France for Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Symkevi (tezacaftor/ivacaftor) for eligible cystic fibrosis patients (For full story click here)
    • Also, new portfolio reimbursement agreement in Italy including Kaftrio, Symkevi, and additional indications of Orkambi and Kalydeco for eligible patients with cystic fibrosis (For full story click here)

Vertex looking to expand its CFTR targeting and gene-editing portfolios

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CI Scientists Commentary:

  • Approved therapies for CF: Kalydeco, Symkevi, Kaftrio, and Trikafta. Current development:

Asset

MOA

Stage of development

VX-561

CFTR stimulant

Phase 2

VX-121 + Tezacaftor + VX-561

CFTR modulator + CFTR stimulant

Phase 2

  • Leveraging multiple partnerships, future development from Vertex is expected to move from targeting CFTR to gene-editing therapies

Partnership with

Technology

Affinia Therapeutics

Affinia proprietary AAV vector technology

Arbor Biotechnologies

Arbor’s proprietary research platform

CRISPR Therapeutics

CRISPR-Cas9 Gene Editing Technology

Moderna

Moderna’s proprietary mRNA and LNP technology

– Dr. Kowndinya, CI Scientists

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