CRISPR, Vertex gain as FDA starts review for gene edited therapy
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CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals (NASDAQ:VRTX) traded higher pre-market Friday after the companies announced that the FDA accepted their Biologics License Applications for gene-edited blood disorder candidate exa-cel.
The companies also posted on Friday pivotal data backing the treatment as scheduled for presentations at the ongoing Annual European Hematology Association (EHA) Congress.
Exa-cel, also known as exagamglogene autotemcel, is an autologous, ex vivo CRISPR/Cas9 gene-edited therapy that has been granted key FDA titles such as Fast Track, Orphan Drug, and Rare Pediatric Disease designations.
The regulator has issued a Priority Review for its sBLA in severe sickle cell disease (SCD) with a target action date on December 8, 2023, and a standard review for that in transfusion-dependent beta-thalassemia (TDT) with a target action date on March 30, 2024.
Announcing updated data from two pivotal trials called CLIMB-111 and CLIMB-121, for exa-cel, ahead of the EHA events, CRISPR (CRSP) and Vertex (VRTX) said that the treatment met primary and key secondary endpoints at a pre-determined interim analysis.
Highlights from the 83-patient readout indicate that out of 27 TDT patients who were evaluable, 24 (88.9%) achieved the primary endpoint of transfusion independence for 12 consecutive months at least.
Out of 17 evaluable patients with SCD, 16 (94.1%) reached the primary endpoint of freedom from vaso-occlusive crises for the same period.
As for safety, two TDT patients developed serious adverse events related to the investigational therapy, as previously disclosed. One adult with SCD died due to COVID-19 in an event unrelated to exa-cel.
Related: Bluebird bio (BLUE) is waiting for the FDA priority review for its gene therapy lovo-cel as a treatment for sickle cell disease.