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ASH: Sickle Cell Treatment Decisions In The Post-Oxbryta, Genetic Medicine Era

Patients Have One Less Option Than A Year Ago

 
• By Mandy Jackson

Hematologists discussed the still limited treatment options for sickle cell disease at ASH, weighing the risks and benefits of disease-modifying versus curative therapies after the withdrawal of Pfizer’s Oxbryta (voxelotor).

Hematologists at ASH lamented the loss of Oxbryta for sickle cell patients (Shutterstock)

This year’s American Society of Hematology (ASH) meeting delivered positive Phase II results for a new daily oral therapy and promising interim Phase I/II data for a one-time base editing treatment for sickle cell disease (SCD). However, the conference was a little less celebratory for the sickle cell field than in 2023, when the annual gathering immediately followed US Food and Drug Administration approval of the first two genetic medicines for SCD.

Key Takeaways
  • Hematologists who treat sickle cell disease already struggle to find effective available treatments for their patients and have one less option after Pfizer withdrew Oxbryta from the market.
  • Doctors at the recent ASH meeting discussed options for SCD patients going forward, including clinical trials and curative therapies, including stem cell transplants and genetic medicines

The Vertex Pharmaceuticals/CRISPR Therapeutics gene-editing treatment Casgevy (exagamglogene autotemcel) and bluebird bio gene therapy Lyfgenia (lovotibeglogene autotemcel) struggled to deliver significant sales in their first year on the market. ASH 2024, from 7-10 December in San Diego, also came less than three months after Pfizer

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