FDA advisers favorable to 1st CRISPR gene therapy

A panel of FDA advisers concluded after hours of discussion Oct. 31 that the first CRISPR gene therapy candidate is safe enough for clinical use, The New York Times reported. 

An advisory committee met Oct. 31 to discuss an experimental drug for sickle cell disease, which would be the first approved therapy made with CRISPR technology if the FDA gives its green light. The agency is expected to make its final decision by Dec. 8. 

CRISPR is a gene-editing tool that stands for clustered regularly interspaced short palindromic repeats, and cancer drug researchers have been investigating its treatment potential for years. 

The candidate is exagamglogene autotemcel, or exa-cel, a drug product developed by Boston-based Vertex Pharmaceuticals and Switzerland-based CRISPR Therapeutics. The drugmakers applied for the drug to be approved for sickle cell disease patients 12 and older.

The Institute for Clinical and Economic Review evaluated exa-cel's cost-effectiveness value between $1.35 million and $2.05 million per dose; the drugmakers have not proposed a list price for the experimental therapy.

Sickle cell disease affects about 100,000 people in the U.S., and the painful blood disorder is most prevalent among people with African and Mediterranean ancestry, according to the CDC and the FDA.

One of the main questions for the advisory panel surrounded exa-cel's "off-target" results, which is when a gene editing therapy slices DNA similar to its intended area. If this happens, a mutation in a critical gene can lead to cancer or other disorders. This risk is small, but the committee is tasked with comparing exa-cel's "off-target" side effects with its therapeutic benefits. 

The panel did not vote on whether to recommend the drug's approval.

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