The Food and Drug Administration’s staff raised concerns about safety data on a potential gene-editing treatment for sickle cell disease, ahead of a closely watched meeting where scientific experts will weigh the first therapy using Crispr technology.
FDA advisers are preparing to meet Oct. 31 to consider and discuss an application from Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG to use a Crispr-based treatment for people with the painful blood disorder.
Crispr makes precisely targeted changes in DNA that researchers are trying to use to repair flaws in patients’ genomes related to inherited disease. It isn’t clear if the genetic diversity of patients studied by the companies in their safety analysis “is sufficient to adequately assess” the risk that the treatment would make unintended changes in patients’ DNA, the FDA staff said Friday in a report, or that it can “adequately capture variants in this population across the United States.”
Shares of Vertex fell 1% as of 12:36 p.m. in New York, while Crispr Therapeutics fell less than 1%.
The report also published data from a clinical trial by the companies that found 93.5% of subjects met the primary goal, which was freedom from pain crisis for 12 months after treatment.
Sickle cell is an inherited disease where patients have red blood cells that are inefficient and misshapen, leading to clogged blood vessels and painful crises when tissues fail to get enough oxygen. The two partners are seeking approval to edit the genes of people with sickle cell disease so that they make a different, oxygen-carrying form of hemoglobin.
Potential Relief
The treatment promises potential relief — and possibly a cure — for the condition that affects some 100,000 Americans, most of whom are Black. The life expectancy of people who have sickle cell disease and are covered by Medicaid and Medicare is about 53 years, according to a study published in July.
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