Catalyst Biosciences recently announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to marzeptacog alfa (MarzAA), the company’s subcutaneous (SQ) factor VIIa therapy. Catalyst is developing MarzAA for the prophylactic treatment of individuals with hemophilia A or B with inhibitors.
“Fast tracking” expedites the review of drugs targeted to treat serious diseases, particularly if they help fulfill the unmet medical needs of patients. Once a drug receives this designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.
According to Catalyst press release, this month MarzAA enters a key phase 3 clinical study known as CRIMSON, a global multicenter trial. It is designed to evaluate the safety and efficacy of the therapy for on-demand treatment of spontaneous or traumatic bleeding episodes, in adolescents and adults with congenital hemophilia A or B with inhibitors, compared to existing inhibitor therapies.
Investigators will enroll approximately 60 subjects to treat 244 eligible bleeding episodes with each treatment. The primary endpoint for the trial is the percentage of treated bleeds resulting in effective hemostasis at the 24-hour timepoint. The CRIMSON study design was presented as a poster at the 62nd Annual American Society of Hematology meeting, held virtually December 5-8, 2020.
“MarzAA is the only SQ delivered therapy in development for on-demand treatment of bleeding events and could become an important addition to the treatment landscape for patients,” said Nassim Usman, PhD, president and chief executive officer of Catalyst. “We’re looking forward to announcing our continued progress.”
Catalyst press release dated December 2, 2020