October 2019 Regulatory Update
The FDA Antimicrobial Drugs Advisory Committee voted 14-2 to recommend approval of cefiderocol for the treatment of complicated urinary tract infections.
The FDA accepted the NDA for rimegepant, for the treatment and prevention of migraines and set a PDUFA date in March 2020.
The FDA accepted the NDA for triheptanoin, for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) and set a PDUFA target date of 7/31/2020.
The FDA accepted the BLA for Trastuzumab deruxtecan, for the treatment HER2-positive metastatic breast cancer and set a PDUFA date in March 2020.
The FDA awarded a Rare Pediatric Disease designation to sepofarsen for the treatment of Leber’s congenital amaurosis 10.
CHMP advised the EMA not to approve quizartinib due to a lack of evidence to support a survival benefit.
Announced Research Updates
Principia announced that in a 24-week, 15 patient, Phase II, open-label trial, 60% of patients treated with PRN1008 achieved CDA and 40% achieved complete remission in patients with moderate to severe pemphigus.
Lilly announced that in the 567 patient, Phase III SEQUOIA trial, the addition of pegilodecakin to FOLFOX (folinic acid, 5-FU, oxaliplatin) did not improve overall survival compared to FOLFOX alone in patients with metastatic pancreatic cancer whose disease had progressed despite a gemcitabine-containing regimen.
UCB announced that in the 16-week, 570 patient, Phase III, BE VIVID trial, more patients treated with bimekizumab achieved a 90% improvement in the Psoriasis Area and Severity Index (PASI 90) and Investigator Global Assessment (IGA) score of clear or almost clear (IGA 0/1) than placebo or ustekinumab in patients with moderate-to-severe chronic plaque psoriasis.
Congo is using both the J&J and Merck vaccines during the 2019 Ebola outbreak.
Published Research Updates
In two Phase III trials (SAKURA 1 and SAKURA 2) a total of 609 patients were treated with daxibotulinumtoxinA and achieved a reduction in their Glabellar line severity score at week 4 by at least two points in 73.6% vs 0% in SAKURA 1 and in 74% vs 1% in SAKURA 2 with line severity not returning to baseline until 27.7 weeks in SAKURA 1 and 26 weeks in SAKURA 2.
In a 223 patient, Phase IIb trial, treatment with GC4419 compared to placebo reduced development of severe radiation-induced oral mucositis (SOM) (43% vs 65%) and duration of SOM (1.5 vs 19 days) in patients with locally advanced oral cavity or oropharynx cancer receiving seven weeks radiation therapy plus cisplatin.
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