Shares of CRISPR Therapeutics AG (NASDAQ: CRSP) surges more than 6% during pre-market trading on Friday. This surge comes in response to an exciting announcement from Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and CRISPR Therapeutics (CRSP). The U.S. Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for the investigational treatment known as exagamglogene autotemcel (exa-cel). The treatment is specifically designed to address severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
The FDA has recognized the importance of these treatment applications by granting Priority Review for SCD and Standard Review for TDT. In addition, the FDA has assigned target action dates for these applications, with December 8, 2023, set as the date for SCD and March 30, 2024, for TDT. The regulatory submissions for the treatment will be backed by updated data from pivotal trials. These significant findings will be showcased at the Annual European Hematology Association Congress on June 11, 2023.
In the United States, exa-cel has already received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations. These designations further emphasize the potential of exa-cel in treating both TDT and SCD.
On the European front, Marketing Authorization Applications (MAAs) for exa-cel were submitted in December 2022 and subsequently validated by the European Medicines Agency (EMA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) in January 2023. The European Commission has granted exa-cel Orphan Drug Designation, and the EMA has provided Priority Medicines (PRIME) designation for both SCD and TDT. In the United Kingdom, exa-cel has also received an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.
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