Breakthrough in CRISPR-Based Therapy: FDA Accepts Vertex and CRISPR Therapeutics' Exa-cel Application

The FDA has accepted the application for Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics AG CRSP exa-cel for sickle cell disease and transfusion-dependent beta-thalassemia (TDT). 

The FDA has granted priority review for exa-cel in SCD with a PDUFA date of December 8, 2023, and standard review for TDT with a PDUFA date of March 30, 2024.

William Blair resumes coverage with an Outperform rating. William Blair analyst says the acceptance of the BLA for exa-cel with priority review for SCD is positive, given it presents a larger market opportunity between the two indications and will be the first CRISPR-based therapy to be reviewed by the FDA for approval. 

It notes the lack of priority review in TDT as unfortunate but does not see it ultimately affecting the probability of exa-cel's approval in TDT or its commercial success. 

The analyst speculates that the standard review process for TDT could be because there is already an FDA-approved gene therapy for TDT from bluebird bio Inc BLUE, decreasing the urgency for novel therapeutics for the indication. 

JMP Securities writes that the exa-cel BLA acceptance by the FDA represents a major milestone ahead of potential approvals that will have read across to all gene editing names. 

It maintains the Market Outperform rating on CRISPR Therapeutics. It increases the price target to $74 from $70 and points to a rapidly approaching regulatory decision in the EU that could be accretive to CRSP shares in the short term.

Truist writes that the news is a significant relief.

Price Action: CRSP shares are down 1.20% at $61.84 on the last check Friday.