US FDA approves Vertex’s CASGEVY™ for the treatment of transfusion-dependent beta thalassemia

The U.S. Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals’ CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.

“On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “TDT patients deserve new, potentially curative treatment options, and we look forward to bringing CASGEVY to eligible patients who are waiting.”

The administration of CASGEVY requires experience in stem cell transplantation; therefore, Vertex is engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to patients.

All nine ATCs activated in the U.S. are able to offer CASGEVY to eligible patients with TDT and sickle cell disease (SCD). Additional ATCs will be activated in the coming weeks.

A message from the Editor:

Thank you for reading this story on our news site - please take a moment to read this important message:

As you know, our aim is to bring you, the reader, an editorially led news site but journalism costs money and we rely on advertising and digital revenues to help to support them.

With the Covid-19 lockdown having a major impact on our industry as a whole, the advertising revenues we normally receive, which helps us cover the cost of our journalists and this website, have been drastically affected.

As such we need your help. If you can support our news sites with a small donation of even £1, your generosity will help us weather the storm and continue in our quest to deliver quality journalism.

In the meantime may I wish you the very best.

- Advertisement -

Related news