▎WuXi AppTec Content Team Editor
A few days ago, Vertex Pharmaceuticals announced that it had completed a discussion with the US FDA and jointly developed with CRISPR Therapeutics, a CRISPR/Cas9-based gene editing therapy exa-cel< strong>Approved by the FDA to submit a rolling application for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Vertex will submit a Biologics License Application for exa-cel through the FDA rolling application process in November this year and is expected to complete the submission by the end of the first quarter of 2023.
Exa-cel (previously known as CTX001) is an investigational autologous in vitro CRISPR/Cas9 gene editing therapy that genetically edits the patient’s own hematopoietic stem cells to make red blood cells produces high levels of fetal hemoglobin (hemoglobin F, HbF) in patients with TDT or SCD.
HbF is a form of oxygen-carrying hemoglobin that occurs naturally during fetal development and is converted to its adult form of hemoglobin immediately after the fetus is born. Elevating HbF levels with exa-cel therapy is expected to reduce the need for blood transfusions in patients with TDT and reduce distress and sickle cell crisis in patients with SCD. These trials are still ongoing, with early results published in the New England Journal of Medicine in January 2021, and updated results presented at the European Association of Hematology (EHA) annual meeting in June.
Vertex has previously completed discussions with the European Medicines Agency (EMA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) on the data needed to support these marketing applications, which are expected to be available by the end of 2022 before submitting this data.
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Based on the progress exa-cel has made to date, the therapy has received a number of important regulatory designations, with the US FDA granting it the treatment of sickle cell disease and transfusion-dependent beta thalassemia A series of regulatory qualifications such as regenerative medicine advanced therapy (RMAT) designation, fast track designation, orphan drug designation and rare pediatric disease designation. In addition, Exa-cel has been granted “Orphan Drug” status by the European Commission and has been granted Priority Medicines (PRIME) designation by the EMA for the treatment of SCD and TDT.
Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer, Vertex, said in a press release: “We are pleased to have completed our pre-submission meeting with regulators on exa-cel and We are delighted that the US FDA has allowed the rolling application. We will continue to work hard to deliver the first CRISPR therapy for an inherited disease with the potential to transform the lives of patients with sickle cell disease and beta thalassemia.”
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