Update for January 11, 2022

Regulatory Update
 
The FDA approved daridorexant (Quviviq, Idorsia), on 1/10/2022, for the treatment of insomnia in adults.
 
The FDA accepted the NDA for tebipenem for the treatment of complicated urinary tract infections and set a PDUFA date for 6/27/2022.
 
Announced Research Updates
 
Biosight announced that in a 66 patients Phase IIb trial, treatment with aspacytarabine resulted in a 37% complete response rate in patients with acute myeloid leukemia who are not eligible for standard induction chemotherapy due to advanced age or comorbidities.
 
BioMarin announced the annualized bleeding rate was maintained at 0.8 per year (0.9 during year one and 0.7 during year two) and Factor VIII infusions at 2.6 per year (1.5 during year one and 3.4 during year two) in 112 patients enrolled in the valoctocogene roxaparvovec GENEr8-1 trial that had been followed for 110 weeks. 
 
Published Research Updates
 
In the 16-week, 215 patient, Phase III, TIMES 3 trial, adding imeglimin to insulin decreased HbA1c 0.6% at 16 weeks and 0.64% at 52 weeks compared to insulin alone in Japanese patients with type 2 diabetes.
 
In the 714 patient, Phase II/III, RELATIVITY-047 trial, patients treated with relatimab plus nivolumab achieved progression-free survival of 10.1 months compared to 4.6 months with nivolumab alone in patients with previously untreated metastatic or unresectable melanoma.
 
In an updated analysis of tirzepatide for the treatment of type 2 diabetes, ICER found that treatment with tirzepatide reduced HbA1c 2.5% and weight by 10.9 Kg when added to background therapy. Analyzing data from a direct comparison with semaglutide and a network meta-analysis with empagliflozin, tirzepatide has a greater decrease in HbA1c, weight loss, lipids, and blood pressure. Treatment with tirzepatide was associated with an increase in gastrointestinal adverse effects, severe adverse events, and discontinuation compared with semaglutide. Because only indirect data was available, a comparison of adverse events with tirzepatide was not possible. While semaglutide and empagliflozin have shown improvement in cardiovascular (CV) outcomes, the tirzepatide CV outcomes trial has not been completed. Due to the lack of the CV outcomes data, ICER found tirzepatide to provide comparable or better net health benefits to semaglutide and empagliflozin. ICER estimated an annual health-benefit price benchmark range of $5,500 to $5,700 for tirzepatide.