The breakthrough therapy status was based on the favourable results of the Phase 2 ENVISION clinical trial in which Sibeprenlimab resulted in a significantly greater decrease in proteinuria than placebo
Otsuka gets FDA breakthrough status for IgAN candidate. (Credit: julien Tromeur on Unsplash)
Otsuka Pharmaceutical and Visterra, an Otsuka group company, have received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for Sibeprenlimab to treat immunoglobulin nephropathy (IgAN) or Berger’s disease.
Sibeprenlimab, formerly known as VIS649, is an investigational humanised IgG2 monoclonal antibody.
It is designed to lower the production of Gd-IgA1 by binding to a specific signalling molecule that drives IgA and Gd-IgA1 production.
IgAN is a common form of primary glomerulonephritis globally and is the most common cause of kidney failure in young adults.
By lowering the production of Gd-IgA1, the investigational drug is believed to stop further kidney damage and the progression to end-stage kidney disease.
The breakthrough therapy status was based on the favourable results of the Phase 2 ENVISION clinical trial. Its results were published in The New England Journal of Medicine.
The multicentre, double-blind, randomised, placebo-controlled, parallel-group trial assessed Sibeprenlimab in biopsy-confirmed IgAN patients.
In the study, the patients received standard-care treatment in a 1:1:1:1 ratio to receive intravenous Sibeprenlimab at a dose of two, four, or eight mg per kilogram of body weight or placebo every month for 12 months.
The primary endpoint was defined as the change from baseline in the log-transformed 24-hour urinary protein-to-creatinine ratio at month 12.
Secondary endpoints consist of the modifications from baseline in the estimated glomerular filtration rate (eGFR) at month 12.
The results showed that Sibeprenlimab resulted in a significantly greater decrease in proteinuria than the placebo.
Additionally, the rate of adverse events that occurred after the start of administration of Sibeprenlimab or placebo was 78.6% in the pooled Sibeprenlimab groups and 71.1% in the placebo group.
Otsuka Pharmaceutical and Visterra are also planning to advance the ongoing Phase 3 trial for Sibeprenlimab for patients with IgAN.