FDA sets Dec deadline for Vertex, CRISPR sickle cell therapy
Vertex is expected to make an approval decision for sickle cell disease by December 8, 2023, and for beta thalassemia by March 30, 2024. results.
In the case of sickle cell disease, an impressive 94% of patients experienced at least one whole year without the excruciating pain caused by blood clot crises
The news emerged as Vertex and CRISPR reported new results from two pivotal trials of the therapy (CLIMB-111 and CLIMB-121), and a longer-term observational study (CLIMB-131), showing that exa-cel continued to meet primary.
Exa-cel is an ‘ex vivo’ application of CRISPR gene-editing, used to modify a patient’s own cells outside the body to make foetal haemoglobin (HbF), which can serve as a substitute to regular haemoglobin in both SCD and beta thalassaemia.
The updated results are due to be reported over the coming weekend at the European Haematology Association (EHA) congress.
Vertex has estimated there are around 32,000 patients in the US who could be eligible for treatment with exa-cel if approved, but hasn’t yet commented on its pricing plans for the therapy, or provided any sales projections.
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