Vertex Continues Gene Therapy Momentum in Blood Disorders With Exa-Cel Submission


Bluebird bio’s ZYNTEGLO was the first gene therapy to be approved in the space in August 2022.

Vertex Pharmaceuticals and CRISPR Therapeutics have announced that they will be submitting biologics license applications (BLA) for exagamglogene autotemcel (exa-cel) for rolling review for the treatments of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) beginning in November 2022.1

Vertex plans to complete the submission by the end of the first quarter of 2023. In the UK and EU, Vertex is on track to submit by the end of 2022.

“We are pleased to have concluded our exa-cel pre-submission meetings with regulators and are excited that FDA has granted a rolling review,” Nia Tatsis, PhD, Executive Vice President and Chief Regulatory and Quality Officer, Vertex, said in a statement.1 “We continue to work with urgency to bring forward the first CRISPR therapy for a genetic disease, and one that holds potential to transform the lives of patients with sickle cell disease or beta thalassemia.”

The news follows in the footsteps of the FDA’s landmark approval of the first gene therapy to be approved for blood disorders: bluebird bio’s betibeglogene autotemcel (beti-cel), now marketed as ZYNTEGLO, for treating TDT.2

READ MORE: Eli-Cel Approved for CALD, Marking Second Gene Therapy Win for bluebird bio

“Today’s approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions,” Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA, said in an FDA statement marking the approval.2 “Given the potential health complications associated with this serious disease, this action highlights the FDA’s continued commitment to supporting development of innovative therapies for patients who have limited treatment options.”

The FDA has granted exa-cel regenerative medicine advanced therapy, fast track, orphan drug (OD), and rare pediatric disease designations for both SCD and TDT.1 The European Commission has granted OD designation and the EMA has granted priority medicines designation to the therapy for both SCD and TDT.

Exa-cel is being investigated in the ongoing phase 1/2/3 open-label CLIMB-111 and CLIMB-121 trials for SCD and TDT, the long-term CLIMB-131 study, and the ongoing, phase 3, open-label CLIMB-141 and CLIMB-151 trials which are currently enrolling participants.1

1. Vertex and CRISPR Therapeutics Announce Global exa-cel Regulatory Submissions for Sickle Cell Disease and Beta Thalassemia in 2022. News release. Vertex Pharmaceuticals. September 27, 2022.
2. FDA approves first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular blood transfusions. News release. FDA. August 17, 2022.
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