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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; ONO 0530

Latest Information Update: 09 Dec 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

  • 06 Dec 2025 Adverse events and efficacy data from a phase IIa trial in Polycythaemia vera released by Deciphera Pharmaceuticals
  • 06 Dec 2025 Deciphera Pharmaceuticals plans a phase III trial for Polycythemia vera by the year 2026
  • 31 May 2025 Sapablursen receives Breakthrough Therapy status for Polycythaemia vera in USA

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