Vertex Pharmaceuticals and CRISPR Therapeutics have completed the rolling submission of biologics licence applications (BLAs) to the US Food and Drug Administration (FDA) for exagamglogene autotemcel (exa-cel) to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

The applications include requests for priority review. If granted, these would shorten the time taken for application review to eight months.

Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. It is being assessed for the treatment of SCD or TDT patients.

The BLAs are supported by data obtained from the ongoing Phase III CLIMB-111 and CLIMB-121 trials, along with an ongoing long-term follow-up CLIMB-131 trial.

The CLIMB-111 and CLIMB-121 trials are designed to evaluate the efficacy and safety of a single exa-cel dose in TDT or SCD patients aged 12 to 35 years.

Vertex Pharmaceuticals chief medical officer and Global Medicines Development and Medical Affairs executive vice-president Carmen Bozic said: “The completion of our exa-cel global regulatory filings is a historic milestone.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

“We want to thank the clinical trial participants and the sickle cell and beta thalassemia communities, as well as the physicians, nurses, coordinators, caregivers and friends who support them.”

The US FDA granted regenerative medicine advanced therapy (RMAT), orphan drug, fast track, and rare paediatric disease designations to exa-cel for the treatment of SCD and TDT.

The European Commission granted orphan drug designation and the European Medicines Agency (EMA) granted priority medicines (PRIME) designation to exa-cel for both the indications.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.