Update for May 18, 2021

Regulatory Update
 
The FDA approved pegcetacoplan (Empaveli, Apellis Pharmaceuticals), on 5/14/2021, for the treatment of paroxysmal nocturnal hemoglobinuria in both treatment naïve patients and patients switching from eculizumab or ravulizumab. Pegcetacoplan was approved with a Black Box warning regarding the increased risk of meningococcal and other serious infections caused by encapsulated bacteria that may become rapidly life threatening or fatal if not recognized and treated early. A Risk Evaluation and Mitigation Strategy (REMS) program requires prescribers to counsel patients on the risk of serious infection, provide patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria.
 
The FDA approved an extended-release, locally administered solution of bupivacaine and meloxicam (Zynrelef, Heron Therapeutics), on 5/13/2021, for postsurgical analgesia after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty.
 
The FDA accepted the NDA for levoketoconazole, for the treatment of endogenous Cushing’s syndrome and set a PDUFA date for 1/1/2022.
 
The FDA granted losmapimod Fast Track status for the treatment of facioscapulohumeral muscular dystrophy.
 
Announced Research Updates
 
Biogen announced that in the 12-month, 50 patient, Phase II/III XIRIUS trial, treatment with cotoretigene toliparvovec did not improve Macular Integrity Assessment (MAIA) Microperimetry compared to an untreated group of male patients with X-linked retinitis pigmentosa.
 
Published Research Updates
 
In a 196 patient, Japanese study, elobixibat was similar to sodium picosulfate  when either drug was added to 1 L of polyethylene glycol formulation containing ascorbic acid as a bowell cleaning preparation for colonoscopy as evidenced by similar Boston Bowel Preparation Scale scores (8.3 vs 8.3). 
 
In the 392 patient, Phase III AHELP trial, treatment with apatinib resulted in overall survival of 8.7 months compared to 6.8 months with placebo in Chinese patients with pretreated advanced hepatocellular carcinoma.
 
In the 52-week, 1,061 patient, Phase III NAVIGATOR trial, the annualized asthma exacerbation rate with tezepelumab was 0.93 compared to 2.1 with placebo in patients 12 to 80 with severe uncontrolled asthma. 
 
In the 16-week, 440 patient, Phase III, BREEZE-AD5 trial, 30% of patients treated with baricitinib 2 mg achieved EASI-75 compared to 8% with placebo in patients with moderate to severe atopic dermatitis. Improvement with a 1 mg dose of baricitinib did not reach significance.
 
In a draft review of JAK inhibits for the treatment of atopic dermatitis, ICER estimated the annual cost of abrocitinib at $37,500 to be cost-effective at $150,000 per QALY. ICER found insufficient evidence to compare abrocitinib to dupilumab. ICER also found that abrocitinib may improve severity, itch and sleep compared to topical therapies. The evidence is considered insufficient to compare abrocitinib, upadacitinib, baricitinib and tralokinumab.
 
In a draft review of JAK inhibits for the treatment of atopic dermatitis, ICER estimated the annual cost of tralokinumab at $31,500 to be cost-effective at $150,000 per QALY. Based on available evidence ICER found the evidence for tralokinumab to be comparable or inferior to dupilumab. ICER also found that tralokinumab may improve severity, itch and sleep compared to topical therapies. Based on indirect evidence, abrocitinib and upadacitinib appear to have more favorable outcomes than baricitinib or tralokinumab.