Posted by Michael Wonder on 21 Dec 2018
FDA approves first treatment for rare blood disease
21 December 2018 - The U.S. FDA today approved Elzonris (tagraxofusp-erzs) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm in adults and in paediatric patients, two years of age and older.
The efficacy of Elzonris was studied in two cohorts of patients in a single-arm clinical trial. The first trial cohort enrolled 13 patients with untreated blastic plasmacytoid dendritic cell neoplasm (BPDCN), and seven patients (54%) achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc). The second cohort included 15 patients with relapsed or refractory BPDCN. One patient achieved CR and one patient achieved CRc.
The FDA granted this application breakthrough therapy and priority review designation. Elzonris also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
