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Sapablursen - Ionis Pharmaceuticals

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Drug Profile

Sapablursen - Ionis Pharmaceuticals

Alternative Names: IONIS TMPRSS6-Lrx; IONIS-TMPRSS6-LRx; ISIS-702843

Latest Information Update: 25 Mar 2025

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At a glance

  • Originator Ionis Pharmaceuticals
  • Class Antineoplastics; Antisense oligonucleotides
  • Mechanism of Action TMPRSS6 protein inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Polycythaemia vera
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Polycythaemia vera
  • Discontinued Beta-thalassaemia; Thalassaemia

Most Recent Events

  • 11 Mar 2025 Sapablursen licensed to Ono pharmaceutical worldwide
  • 07 Nov 2024 Sapablursen - Ionis Pharmaceuticals receives Orphan Drug status for Polycythaemia vera in USA
  • 07 Nov 2024 Ionis Pharmaceuticals completes enrolment in its phase-II clinical trials in Polycythaemia vera in US, Canada, Hungary, Poland, the United Kingdom (SC) (NCT05143957)

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