Update for December 1, 2020

Regulatory Update
 
The FDA approved naxitamab (Danyelza, Y-mAbs Therapeutics), on 11/25/2020, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of relapsed or refractory high-risk neuroblastoma in bone or bone marrow.
 
The FDA approved setmelanotide (Imcivree, Rhythm Pharmaceuticals), on 11/27/2020, for chronic weight management of obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing.
 
The FDA has delayed approval of Revance’s daxibotulinumtoxinA due to travel restrictions delaying the inspection of the manufacturing plant. A new PDUFA date has not been announced.
 
The FDA rejected the NDA for Reata’s omaveloxolone for the treatment of Friedreich’s ataxia and requested additional data to strengthen data from the MOXIe trial.
 
The EMA accepted the MAA for EMD Serono’s tepotinib for the treatment of metastatic non-small cell lung cancer harboring METex14 skipping alterations.
 
Announced Research Updates
 
Immunocore announced interim data from a 378 patient, Phase III trial, where treatment with tebentafusp resulted in estimated overall survival of 73% compared to 58% with investigator choice (82% pembrolizumab; 12% ipilimumab; 6% dacarbazine) in patients with metastatic uveal melanoma.
 
UniQure announced that in the 26-week, 54 patient, Phase III, HOPE-B trial, a single dose of etranacogene dezaparvovec increased Factor IX activity from less than 2% at baseline to 37%.  While 72% of patients reported no bleeding episodes, 15 patients reported 21 bleeding episodes in adult males with severe or moderate-severe Hemophilia B.
 
Urovant announced that in a 12-week, 189 patient, Phase IIa trial, more patients treated with vibegron did not achieve at least a 30% improvement in average worst abdominal pain compared to placebo in women with abdominal pain due to irritable bowel syndrome (IBS) with IBS-D (diarrhea) and IBS-M (mixed IBS).
 
Published Research Updates
 
In a long term extension of the MUSE trial, 218 patients completed a total of 3 years of treatment with anifrolumab and maintained improvements in their SLE Disease Activity Index 2000 (SLEDAI-2K) scores and SLICC Damage Index (SDI) scores.
 
In the 52-week, 455 patient, Phase III, ESAX-DN trial, 22% of patients treated with esaxerenone achieved urinary albumin-to-creatinine ratio remission (< 30 mg/g creatinine and or > 30% reduction) compared to 4% with placebo in Japanese patients with type 2 diabetes and urinary albumin-to-creatinine ratio of 45 to < 300 mg/g creatinine treated with renin-angiotensin system inhibitors.