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Voxelotor: First Approval
1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.
Drugs
|February 6, 2020
Summary
Voxelotor (Oxbryta™) is a novel treatment for sickle cell disease, approved in the USA. This hemoglobin S polymerization inhibitor offers a new disease-modifying option for patients.
Area of Science:
- Hematology
- Pharmacology
Background:
- Sickle cell disease (SCD) is a genetic blood disorder characterized by abnormal hemoglobin.
- Current treatments for SCD focus on managing symptoms and complications.
Purpose of the Study:
- To summarize the development milestones of voxelotor.
- To highlight its first global approval as a disease-modifying agent for SCD.
Main Methods:
- Review of clinical development of voxelotor.
- Analysis of regulatory milestones and trial data.
Main Results:
- Voxelotor (Oxbryta™) received US FDA accelerated approval in November 2019.
- Approval was based on Phase III HOPE trial results for SCD treatment in patients aged ≥12 years.
Conclusions:
- Voxelotor represents a significant advancement in SCD therapy.
- The drug has received Orphan Drug designation and Priority Medicine status in Europe.
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