The Food and Drug Administration (FDA) has granted Orphan Drug designation to PRN1008 (Principia Biopharma) for the treatment of immune thrombocytopenic purpura (ITP).
PRN1008 is an oral, reversible covalent Bruton’s tyrosine kinase (BTK) inhibitor; BTK is present in the signaling pathways of most types of white blood cells except for T cells and plasma cells. The treatment is currently being evaluated in two Phase 2 trials, one in patients with pemphigus vulgaris and one in patients with ITP.
“We believe that PRN1008 has the potential to modulate the immune system’s attack on platelets, the underlying cause of ITP,” said Martin Babler, CEO of Principia. Last year the Company obtained Orphan status for PRN1008 in pemphigus vulgaris.
According to the National Organization for Rare Disorders the incidence of ITP among US adults is estimated at 3.3 per 100,000 adults/year.
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