An oral inhibitor of Bruton’s tyrosine kinase (BTK) together with very low-dose prednisone induces control of disease activity in patients with pemphigus at 12 weeks. This was the shown in the phase 2 Believe-PV proof of concept study .
The current standard of care is high-dose corticosteroids with prednisone-equivalent doses of ≥1 mg/kg with high toxicity and/or rituximab plus moderate-to-high initial doses of corticosteroids (≥0.5-1mg/kg).
“Despite the recent FDA approval of rituximab for moderate-to-severe pemphigus, there remains an unmet need for a quick-acting, steroid-sparing, anti-inflammatory treatment for this rare disease,” said Prof. Dedee Murrell (University of New South Wales, Australia). BTK inhibition interrupts multiple signalling pathways in immune cells, but not in T cells. In this way, B cell function can be impaired without reducing B cells directly. The agent proved also to be effective in naturally occurring pemphigus in do...
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