Drug Profile
Sapablursen - Ionis Pharmaceuticals
Alternative Names: IONIS TMPRSS6-Lrx; IONIS-TMPRSS6-LRx; ISIS-702843Latest Information Update: 30 Jun 2023
Price :
$50
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At a glance
- Originator Ionis Pharmaceuticals
- Class Antineoplastics; Antisense oligonucleotides
- Mechanism of Action TMPRSS6 protein inhibitors
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Phase II Polycythaemia vera
- Discontinued Beta-thalassaemia; Thalassaemia
Most Recent Events
- 03 May 2023 Discontinued - Phase-II for Beta-thalassaemia in Turkey, Thailand, Lebanon, Australia, Greece, USA (SC)
- 06 Apr 2023 Discontinued - Phase-I for Thalassaemia (In volunteers) in Australia (SC) (Ionis Pharmaceuticals pipeline, April 2023)
- 28 Mar 2023 Ionis Pharmaceuticals terminates a phase IIa trial in Beta-thalassaemia in Australia, Lebanon, Thailand, Greece, Turkey (SC), due to favourable safety and tolerability are seen, but efficacy results in the mid-stage study do not meet Ionis' minimum target product profile to justify further development (NCT04059406)