Update for the Week Ending April 4, 2020

Regulatory Update
 
The FDA accepted the BLA for remestemcel-L for the treatment of children with steroid-refractory acute graft versus host disease and set a PDUFA date of 9/30/2020. Remestemcel-L is approved as Temcell in Japan.
 
Blueprint completed a rolling NDA submission for pralsetinib for the treatment of RET fusion-positive non-small cell lung cancer. 
 
Rhythm completed a rolling submission for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity.
 
BMS and Celgene submitted a BLA for idecabtagene vicleucel for the treatment of refractory multiple myeloma.
 
Announced Research Updates
 
Verona announced that in a 40 patient, Phase II, open-label trial, treatment with ensifentrine pressurized metered-dose inhaler increased FEV1 47 mL to 391 mL with doses of 300 mcg to 6000 mcg dose, in patients with moderate to severe COPD.
 
Published Research Updates
 
In a 24-week, 849 patient, Phase III trial, patients treated with tanezumab 5 mg had a greater decrease in the WOMAC pain and physical function scores and the Patient’s Global Assessment of OA (PGA-OA) compared to placebo in patients with osteoarthritis. The 2.5 mg dose of tanezumab had a a greater decrease in the WOMAC pain and physical function scores, but not the PGA-OA compared to placebo. 
 
In the 60 patient, Phase II, open-label, ZUMA 2 trial, a single infusion of KTE-X19 resulted in an overall response rate of 93% in patients with relapsed or refractory mantle cell lymphoma. 
 
In a 24-hour, 151 patient, Phase III, Japanese-registered trial, 55% of patients treated with landiolol achieved a heart rate of 60-94 bpm compared to 33% in the placebo group in patients with sepsis-related tachyarrhythmia.