Dec 6 (Reuters) – Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged 5 to 11 with sickle cell disease escape painful episodes and allowed those with another blood disease that requires frequent transfusions to go without blood transfusions for at least 12 months.
This supports the therapy’s potential to treat blood disorders in younger patient populations and will help Vertex expand the use of Casgevy, which is currently approved for patients 12 years and older with sickle cell disease or transfusion-dependent beta thalassemia (TDT).
“These results are the first clinical data on gene therapy in children aged 5-11 years with SCD and once again demonstrate the transformative potential of Casgevy,” said Carmen Bozic, chief medical officer at Vertex.
Casgevy is based on the Nobel Prize-winning CRISPR gene-editing technology, which uses molecular “scissors” to trim defective parts of genes and then disable or replace them with new, normal DNA strands.
The company will begin filing documents with global regulators in the first half of next year. It has been awarded a Commissioner’s National Priority Voucher for Casgevy in the 5-11 age group, which will speed up scrutiny by the health regulator.
In a late-stage trial, four children with SCD were free of vaso-occlusive crises for at least 12 months after taking Casgevy and with adequate follow-up. The longest period without these incidents was about two years, the company said.
Vaso-occlusive crisis, a hallmark of inherited blood disorders, is caused by sickle red blood cells blocking blood vessels and causing severe and debilitating pain.
Following Casgevy infusion, 12 TDT patients were transfusion-free for a maximum duration of less than two years.
One TDT patient died of pneumonia and multi-organ failure due to severe veno-occlusive disease, a liver disease, related to pre-transplant treatment with the chemotherapy drug busulfan.
The findings were presented at the American Society of Hematology’s annual meeting.
(Reporting by Sripana Roy in Bengaluru; Editing by Alan Barona)