Vertex/CRISPR win FDA approval for gene therapy Casgevy

The US Food and Drug Administration (FDA) has approved Vertex’s CRISPR/Cas9 gene-edited therapy Casgevy (exagamglogene autotemcel [exa-cel]), a few weeks after regulators in the UK.

In November, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization for Vertex and CRISPR Therapeutics’ sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) treatment Casgevy.

Now, the gene-edited therapy has won authorization from the US regulatory body and Casgevy has become the first FDA-approved SCD treatment to use CRISPR/Cas9 technology. The organization said this has signalled “an innovative advancement in the field of gene therapy.”

Last month, Vertex Pharmaceuticals and CRISPR Therapeutics selected UK-based contract development and manufacturing organization (CDMO) RolsinCT – a spinout from the Roslin Institute –  to commercially manufacture the world’s first CRISPR-based gene-edited therapy.

RoslinCT announced it will also be the manufacturing partner of choice following the FDA approval.

The CDMO will use its MHRA commercially approved facility in Edinburgh, Scotland to manufacture Casgevy and the site has already produced over 100 batches of the therapy.

Vertex and CRISPR Therapeutics have collaborated for a number of years, but first partnered on exa-cel in April 2021, with Vertex paying CRISPR $900 million upfront for the commercialization rights. Exa-cel is an autologous, ex vivo CRISPR/Cas9 (a Nobel Prize winning technology) gene-edited therapy in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells.

HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin.

“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, director of the office of therapeutic products within the FDA’s Center for Biologics Evaluation and Research (CBER).

“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”