FDA grants fast track designation to the BTK inhibitor rilzabrutinib for the treatment of patients with immune thrombocytopenia.
The FDA has granted a fast track designation to the BTK inhibitor rilzabrutinib (PRN1008) for the treatment of patients with immune thrombocytopenia (ITP).
“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,” Dolca Thomas, chief medical officer of Principia, a Sanofi company, stated in a press release.
“This is an excellent acknowledgment as we initiate our phase 3 study,” Thomas added. “Fast track designation is designed to facilitate the development and expedite the review of investigational treatments that demonstrate the potential to address unmet needs in serious or life-threatening conditions.”
Rilzabrutinib was designed to possess potential broad utility in immune-mediated diseases and to have an optimized safety and efficacy profile through the use of Principia Biopharma’s Tailored Covalency technology. Because of this technology, the agent should be able to prolong and reverse action at the target site, while being quickly eliminated from the body. Rilzabrutinib was developed to limit its systemic exposure, while could promote fast clinical reversibility of unwanted effects on the immune system.