Update for October 20, 2021

Regulatory Update
 
The FDA rejected daxibotulinumtoxinA due to deficiencies related to the FDA’s onsite inspection at Revance’s manufacturing facility.
 
The FDA rejected narsoplimab due to current data not supporting efficacy in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy.
 
The FDA delayed the PDUFA date for bimekizumab, because of COVID-19 pandemic restrictions preventing inspection of the UCB European manufacturing site for the drug.
 
Sage Therapeutics and Biogen intend to submit NDAs for zuranolone for the treatment of major depressive disorder (MDD) in the second half of 2022 and to treat postpartum depression (PPD) in the first half of 2023.
 
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended approval of abrocitinib for the treatment of atopic dermatitis.
 
Announced Research Updates
 
Biogen announced that in the 28-week, 108 patient Phase III, VALOR trial, treatment with tofersen did not improve the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) compared to placebo in patients with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
 
Entasis announced that in the 28-day, 207 patient, Phase III, ATTACK trial, treatment with sulbactam/durlobactam resulted in 19% mortality compared to 32.3% with colistin in 125 patients with documented Acinetobacter baumannii infection. In a 28-day open-label portion of the trial, where all patients were treated with sulbactam/durlobactam, mortality was 17.9% in patients with Acinetobacter baumannii-calcoaceticus Complex infections that are resistant to or failed colistin or polymyxin B.
 
Dicerna announced that in the six patient, Phase I, PHYOX4 trial, a single dose of nedosiran was well tolerated, but did not result in a 30% or more decrease in 24-hour urine oxalate excretion on at least two consecutive visits over the three-month observation period in patients with primary hyperoxaluria type 3 (PH3).
 
Galera announced that in the 455 patient, Phase III, ROMAN trial, treatment with avasopasem did not decrease the incidence of severe oral mucositis compared to placebo in patients with locally advanced head and neck cancer receiving seven weeks of radiotherapy plus cisplatin.
 
Published Research Updates
 
In the 15-month, 323 patient, Phase III, PLEO-CMT trial, the highest dose of PXT3003 (6 mg baclofen, 0.7 mg naltrexone and 210 mg sorbitol) reduced the Overall Neuropathy Limitation Scale (ONLS) score by 0.37 points more than placebo in patients with mild to moderate Charcot-Marie-Tooth Type 1A Disease.