FDA Highlights: Abatacept shows efficacy for Glomerulosclerosis

by Bruce Sylvester – Abatacept, an FDA-approved treatment of rheumatoid arthritis could become the first targeted therapy for focal segmental glomerulosclerosis glomerulosclerosis (FSGS), a condition that usually causes kidney failure, researchers Massachusetts General Hospital in Boston reported online on Nov. 8  in the New England Journal of Medicine.

The findings were also presented at the American Society for Nephrology annual meeting.

“We identified abatacept as the first personalized, targeted treatment for kidney disease and specifically for FSGS, a devastating and largely untreatable disease,” said lead author Peter Mundel, MD, of the Division of Nephrology in the Massachusetts General Hospital  Department of Medicine. “We also identified a biomarker that helps us discern which patients are most likely to benefit from therapy with abatacept.”

As background, the authors noted that while the underlying disease mechanism is not known, FSGS disrupts podocyte function; podocytes are cells within the glomeruli that are essential to kidney function. Although steroids and some immunosuppressive drugs help some patients, the side effects are problematic.

In all five subjects, abatacept therapy led to remission of FSGS-caused proteinuria. After a single dose of abatacept, two subjects with recurrent disease have remained in remission for three and four years respectively. Two other subjects required a second dose of abatacept when proteinuria reappeared a few weeks after the first dosing, and they have been in remission for 10 and 12 months respectively.

A fifth subject, with high-risk, treatment-resistant disease, who, following abatacept treatment,  went into remission for the first time in over a year, continues in remission a year later. She receives monthly doses of abatacept, but she no longer requires high-dose steroids and immunosuppressive drugs.

“We have a decade of good experience with the use of abatacept for rheumatoid arthritis, so we have every reason to believe that it will be an excellent long-term option for the treatment of all B7-1-positive diseases, including FSGS and perhaps diabetic kidney disease, ”  Mundel added.