" class="no-js "lang="en-US"> FDA Accepts Applications for Sickle Cell and Thalassemia Drug
Wednesday, May 22, 2024

FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Vertex Pharmaceuticals and CRISPR Therapeutics today announced that the United States Food and Drug Administration (FDA) has accepted the Biologics License Applications for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The FDA has granted Priority Review for SCD and Standard Review for TDT and assigned Prescription Drug User Fee Act (PDUFA) target action dates of December 8, 2023, and March 30, 2024, respectively. Updated data from the pivotal trials supporting the regulatory submissions will be presented at the Annual European Hematology Association Congress on June 11, 2023.

“We are very pleased with the acceptance of the submissions and the Priority Review designation for SCD by the FDA, as well as the progress of the exa-cel filings in the EU and U.K.,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “Exa-cel holds the promise to be the first CRISPR gene-editing therapy to be approved, and we continue to work with urgency to bring this treatment with transformative potential to patients who are waiting.”

“We are glad to see that the unmet need and urgency for innovative therapies in SCD was recognized by the FDA with Priority Review,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “This is an exciting milestone for the CRISPR platform, and we look forward to continuing the close collaboration with our partners at Vertex to bring this medicine to patients in need.”

In the U.S., exa-cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA for both TDT and SCD.

In Europe, the Marketing Authorization Applications (MAAs) for exa-cel were submitted in December 2022 and validated by the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) in January 2023. In the EU, exa-cel has been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the EMA, for both SCD and TDT. In the UK, exa-cel has also been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.

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