Advisers will consider whether more research is needed into potential unintended consequences of the new therapies<\/p>\n<\/h3>\n
By Physician\u2019s Briefing Staff HealthDay Reporter<\/b><\/p>\n
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MONDAY, Oct. 30, 2023 (HealthDay News) — Patients with sickle cell disease may soon have two new treatments to try. On Tuesday, a U.S. Food and Drug Administration advisory committee will weigh the merits of a new gene therapy for the painful, inherited condition. The agency is expected to make a decision on that therapy in early December, and it also plans to decide on a second new treatment before year’s end, the Associated Press<\/em> reported.<\/p>\n The treatment being reviewed<\/a> Tuesday is based on CRISPR technology, a gene-editing tool. The inventors of that tool won the Nobel Prize in 2020 for their work, the AP<\/em> reported. A one-time treatment, “exa-cel” is made by Vertex Pharmaceuticals and CRISPR Therapeutics, and it permanently changes DNA in a patient’s blood cells.<\/p>\n On Tuesday, FDA advisers will consider whether more research is needed into potential unintended consequences of the new gene therapy.<\/p>\n In briefing documents<\/a> filed with the advisory committee, Vertex said that 46 people got the treatment in its study. Among the 30 who had 18 months of follow-up, 29 were free of pain crises for at least a year and all 30 avoided being hospitalized for pain crises. Still, the FDA advisory panel is asking outside gene therapy experts to discuss the possibility of “off-target effects” (i.e., unexpected changes to a person’s genome). The FDA would like to determine whether company research on these possible effects has been adequate or whether more studies are needed, the AP<\/em> reported. The company has proposed a postapproval safety study and product labeling that notes potential risks.<\/p>\n The second gene therapy for sickle cell disease that the FDA will consider is intended to work by making functional copies of a modified gene, the AP<\/em> reported. This helps red blood cells produce hemoglobin that is not misshapen. That treatment is made by Bluebird Bio.<\/p>\n Prices for the two gene therapies have not been released, the AP<\/em> reported. However, a price tag of around $2 million would be considered cost-effective because the existing treatments cost about $1.6 million for women and $1.7 million for men from birth to age 65 years, according to recent research<\/a>.<\/p>\n