Principia Biopharma Inc., a clinical-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology, today announced that PRN1008, an oral, reversible covalent Bruton’s Tyrosine Kinase (BTK) inhibitor, has been granted orphan-drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with immune thrombocytopenic purpura (ITP). Principia is currently conducting a Phase 2 clinical trial to evaluate the safety and efficacy of PRN1008 in patients with ITP. Principia previously received orphan-drug designation for PRN1008 by the FDA for the treatment of pemphigus vulgaris in June 2017.
“The FDA granting orphan-drug designation is an important milestone as we continue to develop PRN1008 for ITP patients in need,” said Martin Babler, Chief Executive Officer of Principia. “We believe that PRN1008 has the potential to modulate the immune system’s attack on platelets, the underlying cause of ITP, and to be an effective oral therapy to reduce the untoward effects often seen with more invasive or broader immune suppression.”
- From Principia Biopharma Inc. press release