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Nov. 25 Company Quick Takes: FDA approves GBT's sickle cell therapy; plus ChemoCentryx, Astellas, La Jolla, Sorrento, Aquestive

November 26, 2019 1:06 AM UTC
Updated on Nov 26, 2019 at 8:12 PM UTC

GBT's sickle cell therapy snags accelerated approval
FDA granted accelerated approval to Oxbryta voxelotor from Global Blood Therapeutics Inc. (NASDAQ:GBT) to treat sickle cell disease three months ahead of its Feb. 26 PDUFA date, marking the second new therapy approved for the indication this month. The oral allosteric modifier of hemoglobin oxygen affinity, which was approved based on improvements in hemoglobin levels, will have a wholesale acquisition cost (WAC) of $10,417 per month. Adakveo crizanlizumab-tmca from Novartis AG (NYSE:NVS; SIX:NOVN) was approved for SCD this month based on reductions in the frequency of vaso-occlusive crises for sickle cell patients and has a WAC of $7,071-$9,428 per month (see "Sickle Cell Disease Poised for Fresh Infusion of Hemoglobin-based Endpoints").

ChemoCentryx soars on Phase III vasculitis data
ChemoCentryx Inc. (NASDAQ:CCXI) climbed $25.44 (316%) to $33.50 in after-hours trading Monday after reporting that avacopan met both primary endpoints of the Phase III ADVOCATE trial to treat patients with anti-neutrophil cytoplasmic antibody-associated vasculitis. The small molecule complement receptor 5a inhibitor showed non-inferiority in remission rates compared with glucocorticoid standard of care at 26 weeks and achieved superiority on the same measure at 52 weeks. The candidate also led to reductions in glucocorticoid-related toxicity, improvements in kidney function and improvements in quality of life. ChemoCentryx is planning regulatory submissions in the U.S. and Europe for 2020. ...