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ADVANCED THERAPIES | Vertex, CRISPR To Submit Exa-Cel to FDA For β-Thalassaemia And Sickle Cell Disease

Vertex Pharmaceuticals and CRISPR Therapeutics announced yesterday that Vertex has concluded discussions with the U.S. Food and Drug Administration (FDA), and the FDA granted exagamglogene autotemcel (exa-cel), formerly known as CTX001™, a rolling review for the potential treatment of transfusion-dependent β-thalassaemia (TDT) and sickle cell disease (SCD).

Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022 and expects to complete the submission package by the end of Q1 2023.

Vertex has also completed discussions with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) on the data required to support those marketing applications and 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,” said Nia Tatsis Ph.D., Executive Vice President, Chief Regulatory and Quality Officer. “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.”

If approved, exa-cel would become the first marketed therapy based on the CRISPR gene editing technology that won a Nobel Prize in 2020. Data generated in clinical studies have so far shown that, for most patients, a one-time treatment with exa-cel significantly alleviates the symptoms and burdens of sickle cell and β-thalassaemia.

Based on progress in this program to date, exa-cel has been granted multiple important regulatory designations, including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease Designations from the FDA for both SCD and TDT. Exa-cel has also been granted Orphan Drug Designation (ODD) from the European Commission, as well as Priority Medicines (PRIME) designation from the EMA, for both SCD and TDT.

Εxa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy in which a patient’s own hematopoietic stem cells are edited to produce high levels of fetal haemoglobin (HbF; haemoglobin F) in red blood cells.

The therapy is currently being studied in five different clinical trials. CLIMB‑111 and CLIMB‑121 are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 12 to 35 years, and CLIMB‑141 and CLIMB‑151 aim to assess the same in patients ages 2 to 11 years. The final trial, CLIMB-131, will study the effects of the other four trials on participants for up to 15 years after exa-cel infusion.

Read Vertex’s Full Press Release HERE.

Read More About exa-cel HERE.


Image Credit: Jennifer Cook-Chrysos/Whitehead Institute

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