Vertex Announces New Drug Submission For Exagamglogene Autotemcel Has Been Accepted For Priority Review By Health Canada For The Treatment Of Sickle Cell Disease And Transfusion-Dependent Beta Thalassemia
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Vertex Pharmaceuticals (NASDAQ:VRTX) announced its New Drug Submission for exagamglogene autotemcel (exa-cel) has been accepted for Priority Review by Health Canada for treating sickle cell disease and transfusion-dependent beta thalassemia in patients aged 12 and older. This marks the first CRISPR-based gene-edited therapy submitted for review in Canada. The review timeline is reduced to 180 days from the conventional 300 days, supported by positive Phase 3 study results.
April 01, 2024 | 3:01 pm
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Vertex Pharmaceuticals' exa-cel, a CRISPR-based therapy for sickle cell disease and beta thalassemia, has been accepted for Priority Review by Health Canada, indicating a significant step towards commercialization in Canada.
The acceptance of exa-cel for Priority Review by Health Canada is a critical regulatory milestone for Vertex Pharmaceuticals, potentially accelerating the commercial availability of this innovative therapy in Canada. Given the groundbreaking nature of CRISPR-based gene editing for treating serious genetic diseases, this development could significantly enhance Vertex's market position and investor perception, likely leading to a positive short-term impact on VRTX's stock price.
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