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September 5, 2025Clinical Pharmacology & TherapeuticsOpen Access

Safety, Pharmacokinetics, and Pharmacodynamics of Osivelotor for Sickle Cell Disease: First‐in‐Human Studies in Healthy Participants and Patients

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Authors

ELEleanor LisbonRBR. Clark BrownARAndrew Redfern

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Overview

Phase 1 trials revealed osivelotor improved Hb concentration in sickle cell disease patients, suggesting its potential as an effective treatment.

Key Points

  • Osivelotor treatment demonstrated improvements in Hb concentration and RBC deformability, suggesting meaningful pharmacodynamic activity.
  • The terminal elimination half-life of osivelotor in sickle cell disease participants was notably shorter at approximately 10 days.
  • Safety data indicated osivelotor was well tolerated across healthy participants and those with sickle cell disease without treatment discontinuation.
  • Dose-dependent pharmacokinetics supported the development of loading and maintenance dosing regimens for optimal therapeutic effects.

Cite This Study

Lisbon et al. (2025) studied this question.

synapsesocial.com/papers/68bb5f266d6d5674bcd03053https://doi.org/10.1002/cpt.70028
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1A Phase 1 Microtracer Study Evaluating the Mass Balance, Excretion, and Pharmacokinetics of Osivelotor in Healthy Participants2026
  2. 2Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease2019 · 100 citations
  3. 3Over 4 Years of Safety and Efficacy with Voxelotor in Patients with Sickle Cell Disease2024 · 1 citations
  4. 4Optimization of stem cell fitness and mobilization using moderate transfusion and an oral anti-sickling agent in the sickle mouse model.2025
  5. 5Targeting hemolysis and raas: Osivelotor and losartan ameliorate sickle cell renal pathology2025