FDA Approvals Roundup: Besremi
A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).New approval
Besremi okayed for rare blood disease
PharmaEssentia’s Besremi (ropeginterferon alfa-2b-njft injection) has been approved for treating adults with polycythemia vera, a blood disease caused by a JAK2V617F mutation that results in overproduction of red blood cells (RBCs).
The monopegylated, long-acting interferon alfa-2b reduces excess RBCs and can maintain normal RBC levels for at least a year. The disease is also treated through phlebotomy, a procedure that removes excess RBCs though a cannula inserted into a vein.
Approval of the biologics license application for Besremi was based on efficacy findings from the multicenter, single-arm PEGINVERA trial in which 51 patients from the indicated population received the study drug for an average of 5 years. Overall, 61% of patients had a complete hematological response (CHR), defined as RBC volume, <45%, without a recent phlebotomy; normal white cell and platelet counts; normal spleen size; no blood clots. Among those achieving CHR, time to response was 7.8 months of treatment and median duration of response (DoR) was 14.3 months. In all, 80% of patients achieved a hematological response, based only on hematocrit, platelets, and leukocytes, with a median DoR of 20.8 months.
Besremi received orphan drug designation for this indication.
The drug was approved with a boxed warning for risk of serious disorders, including aggravation of neuropsychiatric, autoimmune, ischemic, and infectious disorders.
