US FDA approval tracker: November 2020

Last month was big on FDA knockbacks, with the coronavirus pandemic playing a major part. Five complete response letters were disclosed due to chemistry, manufacturing and control issues, and there were two Pdufa delays as travel restrictions hampered manufacturing inspections. The new Pdufa date for one of the delayed projects, Bristol’s lisocabtagene maraleucel, has not yet been announced and the company is edging ever closer to the CVR deadline of December 31. On the approval side, Alnylam’s Oxlumo was given the green light a week early; it is used to treat primary hyperoxaluria type 1, a rare genetic disease. Another rare disease player, Rhythm, received its first ever approval: Imcivree gained the thumbs up in pro-opiomelanocortin deficiency obesity and leptin receptor deficiency obesity. However, only around 30 patients have been identified and Rhythm does not plan to deploy a salesforce yet. Phase III data are due soon in the bigger indication of Bardet-Biedl syndrome and there are plans for a basket trial to expand into additional genetically defined obesities. Three emergency use authorisations were granted for Covid-19 treatments last month, but all eyes are on December when two vaccines could get FDA backing.