Update for September 8, 2020

Regulatory Update
 
The FDA approved a bioresorbable collagen implant that contains bupivacaine (Xaracoll, Innocoll), on 8/31/2020, for acute postsurgical pain relief following open inguinal hernia repair.
 
The FDA approved somapacitan (Sogroya, Novo Nordisk), on 9/2/2020, for adults with growth hormone deficiency.
 
The FDA approved pralsetinib (Gavreto, Roche), on 9/4/2020, for the treatment of metastatic RET fusion–positive non–small cell lung cancer (NSCLC), as detected by an FDA-approved test.
 
The FDA rejected Mezzion’s udenafil, in August 2020, as a treatment of single ventricle heart disease and requested additional details and documentation.
 
The FDA accepted the NDA for CorMedix’s taurolidine as a catheter lock solution in hemodialysis patients for the prevention of catheter related blood stream infections and set a PDUFA date for 2/28/2021.
 
The FDA accepted the NDA for Aurinia Pharmaceuticals’ voclosporin, for the treatment of lupus nephritis, and set a PDUFA date for 1/22/2021.
 
The FDA accepted the NDA for Ascendis Pharma’s lonapegsomatropin, for the treatment of pediatric growth hormone deficiency, and set a PDUFA date for 6/25/2021.
 
The FDA designated Mustang Bio’s MB-107 an Orphan Drug for the treatment of X-linked severe combined immunodeficiency (bubble boy disease).
 
Announced Research Updates
 
J&J discontinued development of pimodivir, for the treatment for influenza A infection, after an interim analysis of a Phase III trial in hospitalized patients with influenza, did not find a benefit compared to placebo. J&J also discontinued a Phase III trial evaluating pimodivir in the treatment of influenza A in non-hospitalized patients. 
 
Akebia announced that in the 36-week, 1,751 patient, Phase III, PRO2TECT Correction trial, vadadustat was non-inferior to darbepoetin in the change in hemoglobin (0.05 g/dL difference) and hemoglobin level (10.35 vs 10.39 g/dL) in CKD patients with anemia not on dialysis, who had not received previous ESA treatment. In the 36-week, 1,725 patient, Phase III, PRO2TECT Conversion trial, vadadustat was non-inferior to darbepoetin in the change in hemoglobin (0.01 g/dL difference) and hemoglobin level (10.77 vs 10.77 g/dL) in CKD patients with anemia not on dialysis, who had been receiving an ESA. The PRO2TECT Correction and Conversion trials included 3,471 CKD patients with anemia not on dialysis and found that vadadustat caused more major adverse cardiovascular events than darbepoetin (HR 1.17).
 
Published Research Updates
 
In the 509 patient, Phase III, BROCADE3 trial, veliparib added to carboplatin and paclitaxel improved progression-free survival at 2-years to 14.5 months compared to 12.6 months without veliparib in patients with HER2-negative advanced breast cancer and a germline BRCA1 or BRCA2 mutation.
 
In a 12-month, 286 patient, Phase IIb, open-label, dose-ranging trial, treatment with avacincaptad pegol reduced geographic atrophy growth by 27.4% with 2 mg and 27.8% with 4 mg compared to placebo in patients with geographic atrophy secondary to dry age-related macular degeneration.
 
In a 6-month, 105 patient, Phase II trial, treatment with vupanorsen lowered triglyceride 47% with weekly 20 mg, 36% with 40 mg every four weeks, and 53% with 80 mg every four weeks compared to a 15% decrease with placebo in patients with hypertriglyceridemia, type 2 diabetes and non-alcoholic fatty liver disease.
 
In the 24-week, 177 patient, Phase II/III, CENTAUR trial, treatment with AMX0035 (a combination of sodium phenylbutyrate and taurursodiol) resulted in a 1.24 point decrease per month in functional status (Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised - ALSFRS-R) compared to a 1.66 month decrease with placebo. Most patients in the trial were receiving riluzole, edaravone, or both.