Accelerated drug approvals and patient trust: impact of voxelotor and crizanlizumab for sickle cell disease

Date
2025
Language
American English
Embargo Lift Date
Committee Members
Degree
Degree Year
Department
Grantor
Journal Title
Journal ISSN
Volume Title
Found At
American Society of Hematology
Can't use the file because of accessibility barriers? Contact us with the title of the item, permanent link, and specifics of your accommodation need.
Abstract

Crizanlizumab and voxelotor were several of the first drugs to receive the US Food and Drug Administration (FDA) approval for sickle cell disease (SCD) since the approval of hydroxyurea in 1998. Although initially exciting, additional data regarding efficacy and safety have since emerged for both drugs, first with the removal of crizanlizumab from the European market in August 2023. This was followed by Pfizer's abrupt decision in September 2024 to pull voxelotor from global markets owing to higher mortality in those on the drug vs placebo. These drugs highlight the importance and limitations of the FDA's accelerated approval process. In addition, the impact of these events, without transparent messaging, potentially threatened the fragile trust that providers have more recently been able to build with the SCD population regarding the medical system and research. Although there is a need for new therapies in SCD, we must prioritize both safety and efficacy and maintain trust in this population.

Description
item.page.description.tableofcontents
item.page.relation.haspart
Cite As
Karkoska K, Jacob SA, McGann PT. Accelerated drug approvals and patient trust: impact of voxelotor and crizanlizumab for sickle cell disease. Blood Adv. 2025;9(11):2857-2862. doi:10.1182/bloodadvances.2025015822
ISSN
Publisher
Series/Report
Sponsorship
Major
Extent
Identifier
Relation
Journal
Blood Advances
Source
PMC
Alternative Title
Type
Article
Number
Volume
Conference Dates
Conference Host
Conference Location
Conference Name
Conference Panel
Conference Secretariat Location
Version
Final published version
Full Text Available at
This item is under embargo {{howLong}}