Update for November 1, 2022

Regulatory Update
 
The FDA approved tremelimumab (Imjudo, AstraZeneca) in combination with durvalumab, on 10/24/2022, for the treatment of unresectable hepatocellular carcinoma.
 
The FDA approved teclistamab (Tecvayli, Janssen), on 10/25/2022, for the treatment of relapsed or refractory multiple myeloma in patients who previously received four or more prior lines of therapy, including a proteasome inhibitor, immunomodulatory drug and anti-CD38 monoclonal antibody. Teclistamab was approved with a boxed warning for Cytokine Release Syndrome (CRS) and Neurologic Toxicity.
 
The FDA rejected bulevirtide due to concerns regarding the manufacture and delivery of the drug.
 
Daprodustat

• An FDA review found that daprodustat increased hemoglobin levels similar to erythropoiesis-stimulating agents (ESAs) in the ASCEND-D and ASCEND-ND trials. In dialysis-dependent CKD patients the review noted an increased risk for hospitalization for heart failure and bleeding gastric erosions, but not an increase in major adverse cardiac events. In non-dialysis-dependent CKD patients the review noted an increased risk for hospitalization for heart failure, bleeding gastric erosions, and major adverse cardiac events. Since ESAs already have warnings for some of these risks, the FDA found a further increase to be concerning.
  

• The FDA’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted 13-3 to recommend approval of daprodustat in the treatment of anemia in chronic kidney disease in patients on dialysis. But, the CRDAC voted 11 to 5 not to recommend approval in non-dialysis CKD. The CRDAC members also expressed concern about long-term safety and wanted to see more data.
  

• The FDA has previously rejected vadadustat and roxadustat, two other hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHI), for both dialysis and non-dialysis CKD due to safety concerns.
  

An FDA review of omburtamab found that an external comparator population used in a study was not well matched to the study population. The FDA also felt the data provided in the omburtamab BLA did not demonstrate an improvement in overall survival or support efficacy.
 
The FDA accepted the NDA for quizartinib for the treatment of acute myeloid leukemia (AML) that is FLT3-ITD positive and set a PDUFA date for 4/24/2023.
 
The FDA accepted the BLA for SER-109 for the prevention of recurrent C. difficile infection and set a PDUFA date for 4/26/2023.