Rilzabrutinib granted FDA Fast Track Designation for treatment of immune thrombocytopenia

• Phase 3 trial initiated to evaluate rilzabrutinib, the potential first BTK inhibitor (Bruton’s tyrosine kinase inhibitor) for the treatment of immune thrombocytopenia
• Rilzabrutinib previously granted FDA orphan drug designation

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to the oral investigational Bruton’s tyrosine kinase (BTK) inhibitor, rilzabrutinib, which has the potential to be the first BTK inhibitor for the treatment of immune thrombocytopenia (ITP). In addition, following positive Phase 1/2 study results, a Phase 3 study evaluating rilzabrutinib for ITP has been initiated. Rilzabrutinib received orphan drug designation from the FDA for the treatment of ITP in October 2018.

"By awarding Fast Track Designation to rilzabrutinib, an investigational candidate for the treatment of ITP, the FDA has recognized rilzabrutinib's potential to meaningfully improve outcomes for patients with this debilitating disease. This is an excellent acknowledgement as we initiate our Phase 3 study,” said Dolca Thomas, Chief Medical Officer of Principia, a Sanofi (NYSE: SNY) company. “FTD is designed to facilitate the development and expedite the review of investigational treatments that demonstrate the potential to address unmet medical needs in serious or life-threatening conditions.”