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Following the FDA's recent green light for the Casgevy gene therapy, approval of a similar product is “unlikely” to occur “in the near term,” according to a report from GlobalData.
According to GlobalDataonly 24 CRISPR-based drugs are in phase 2 trials, with 88% of such drugs in an earlier or preclinical stage of development.
CRISPR and its partner Vertex Pharmaceuticals (VRTX) made history earlier this month when Casgevy became the first FDA-approved treatment using CRISPR gene editing technology. The product was approved for the treatment of sickle cell anemia and is pending approval for transfusion-dependent thalassemia.
“The Casgevy approvals represent a significant milestone for revolutionary genome editing systems,” said Jasper Morley, Drug Intelligence Analyst at GlobalData. “However, given the relative immaturity of the CRISPR drug pipeline, featuring very few late-stage products along with a low probability of approval, it is unlikely that another drug will be approved in the near future.”
In its report, published earlier this week, GlobalData listed CRISPR Therapeutics (NASDAQ:CRSP) drug candidate CTX-110 as the next CRISPR-based gene therapy likely to launch globally, possibly towards the end of 2025.
That estimate appears to be based on the fact that CTX-110 was further along than most other CRISPR-related prospects, having reached Phase 2 development for B-cell malignancies. However, even GlobalData gave the product a relatively slim chance of eventually reaching the market, saying it had less than a 50/50 chance of a Phase 3 trial and only a 31% chance of approval.
Meanwhile, in early December, CRSP announced that it would shift its focus from CTX-110 and another prospect, CTX-130, to other potential therapies, namely CTX-112 and CTX-131.
“We are very encouraged by the progress and early clinical data of our next-generation candidates. While we saw benefits from consolidation dosing with CTX110, we believe CTX112 could lead to even better outcomes for patients,” said PK Morrow, chief medical officer. from CRSP. he said in a Dec. 4 press release.
Editor's note: The framing of this story has been changed from its original version to more accurately describe the likely timeline for future CRISPR approvals. The story has been updated to provide significant additional context about CTX-110, including the channel update provided by the company earlier this month.
