Update for July 27, 2021

Regulatory Update
 
The FDA approved odevixibat (Bylvay, Albireo Pharma), on 7/20/2021, for the treatment of progressive familial intrahepatic cholestasis (PFIC) pruritus.
 
The FDA approved fexinidazole, on 7/19/2021, for the treatment of Human African trypanosomiasis in patients 6 years of age and older and weighing at least 20 kg.
 
The FDA rejected retifanlimab and requested additional data to demonstrate a clinical benefit in the treatment of patients with locally advanced or metastatic squamous cell carcinoma of the anal canal.
 
The FDA rejected sulopenem etzadroxil/probenecid, for the treatment of uncomplicated urinary tract infections (UTI) resistant to quinolones. The FDA also requested an additional trial using a different comparator antibiotic. 
 
The FDA delayed approval of Novartis’ inclisiran, in December 2020, because travel restrictions delayed the inspection of the manufacturing plant. Novartis was in the process of moving manufacturing to its own plant in Austria from an Italian plant run by contractor Corden Pharma, when it received the rejection. Novartis completed the transfer and refilled an NDA in July 2021, listing the Novartis owned site for manufacturing. A new PDUFA date was set for 1/1/2022.
 
The FDA delayed the PDUFA date for abrocitinib. A new date has not been announced.
 
The European Commission approved elivaldogene autotemcel (Skysina/Lenti-D, Bluebird Bio) to treat early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen matched sibling hematopoietic stem cell donor is not available.