Press release
Vertex and CRISPR Therapeutics Complete Submission of Rolling Biologics License Applications (BLAs) to the US FDA for exa-cel for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
-EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023- BOSTON & ZUG,
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n-EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023-\n\n\n BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced the completion of the rolling Biologics License Applications (BLAs) to the U.S. Food and Drug Administration (FDA) for the investigational treatment exagamglogene autotemcel (exa-cel) for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The BLAs include requests for Priority Review, which if granted, would shorten the FDA’s review of the application to eight months from the time of submission versus a standard review timeline of 12 months.\n\nThis press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20230402005036/en/\nThese submissions are supported by results from the ongoing Phase 3 studies, CLIMB-111 and CLIMB-121, as well as an ongoing long-term follow-up study, CLIMB-131. Data from the Phase 3 studies were most recently presented at the American Society of Hematology (ASH) Annual Meeting and Exposition in December of 2022.\n\n\n“The completion of our exa-cel global regulatory filings is a historic milestone,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex Pharmaceuticals. “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.”\n\n\n“Within a decade, we have progressed from the discovery of the CRISPR platform to the first regulatory filings for a CRISPR-based therapy, which speaks to the transformative nature of CRISPR technology,” said Phuong Khanh (P.K.) Morrow, M.D., FACP, Chief Medical Officer at CRISPR Therapeutics.\n\n\nIn 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 SCD and TDT.\n\n\nIn 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 Janu...