Update for April 6, 2021

Regulatory Update 
 
The FDA approved viloxazine (Qelbree, Supernus Pharmaceuticals), on 4/2/2021, for the treatment of attention-deficit hyperactivity disorder in patients 6 to 17.
 
Three members of the FDA Peripheral and Central Nervous System Drugs Advisory Committee, which reviewed and rejected aducanumab, published an editorial in JAMA to explain why the drug should not be approved. The authors felt that post hoc analysis should not discredit the negative study, since no other trial based on reduction of beta-amyloid has shown a benefit. They also questioned whether the FDA worked too closely with the study sponsor in a reanalysis of the trials. In conclusion, the authors did not feel that a single positive study justified the approval of aducanumab.
 
Mezzion re-submitted an NDA for udenafil as a treatment to improve exercise capacity in patients with single ventricle heart disease who have undergone a Fontan operation. 
 
AbbVie announced that the FDA accepted the NDA for atogepant as prophylaxis of episodic migraine. AbbVie expects a PDUFA date in late 3Q21.
 
Akebia submitted an NDA for vadadustat for the treatment of anemia due to chronic kidney disease in both dialysis dependent and non-dependent patients.
 
Amryt submitted an NDA for oleogel-S10 for the treatment of the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa.
 
BeyondSpring submitted an NDA for plinabulin plus granulocyte colony-stimulating factor for the prevention of chemotherapy-induced neutropenia.
 
Johnson & Johnson and Legend Biotech have completed a rolling NDA for ciltacabtagene autoleucel as a treatment for relapsed and/or refractory multiple myeloma.
 
CTI BioPharma has submitted an NDA for pacritinib for the treatment of patients with myelofibrosis with severe thrombocytopenia.
 
Announced Research Updates
 
Revance announced that in a 36-week, 301 patient, Phase III trial, daxibotulinumtoxinA improved the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) at six-weeks by 12.7 points with 125 units and 10.9 points with 250 units compared to a 4.3 point improvement with placebo in patients with moderate to severe cervical dystonia. There was no statistical difference between doses in the TWSTRS score. The median duration of effect (loss of 80% of improvement) with 250 units.
 
Published Research Updates
 
In the 24-week, 106 patient, Phase II, PULSAR trial, treatment with sotatercept reduced pulmonary vascular resistance more than placebo by 145.8 dyn·sec·cm(−5) with 0.3 mg/kg and 239.5 dyn·sec·cm(−5) with 0.7 mg/kg in patients with pulmonary arterial hypertension. 
 
In a 355 patient, open-label, Phase III, Chinese trial, progression-free survival (PFS) was 7.6 months with tislelizumab added to paclitaxel and carboplatin and 7.6 months with tislelizumab plus nab-paclitaxel and carboplatin compared to 5.5 months with paclitaxel and carboplatin alone in patients with squamous non-small cell lung cancer. 
 
In the three-month, 114 patient, Phase III COMPASS trial, patients treated with volanesorsen had a 71.2% decrease in triglycerides compared to a 0.9% decrease with placebo in patients with multifactorial severe hypertriglyceridaemia or familial chylomicronaemia syndrome, who had a BMI of 45 kg/m2 or less and fasting plasma triglyceride of 500 mg/dL or higher.
 
In a seven-day, 34 patient, cross-over, Phase II trial, 52% of patients treated with velusetrag achieved at least a 20% reduction in the gastric emptying half-time compared to 5% with placebo in patients with diabetic or idiopathic gastroparesis.