National Pharmacy Week: October 17–23, 2021
National Pharmacy Week acknowledges the invaluable contributions pharmacists and technicians make to patient care in hospitals, outpatient clinics, and other healthcare settings. Celebrated the third full week in October, “Pharmacy Week” is a time to recognize the powerful impact you make on your patients. It is an opportunity to raise your patients’ and colleagues’ awareness about the vital role pharmacists play on the healthcare team. It also provides a meaningful way for you to affirm your staff's hard work to ensure medication use at your organization is safe and patients are achieving optimal outcomes. Regulatory Update The FDA rejected elamipretide for the treatment of Barth syndrome and requested a well-controlled trial. The FDA found that in the Phase II trial submitted for approval, the blinded portion was negative, and the positive open-label extension was not adequately controlled to determine efficacy. Announced Research Updates LEO Pharma announced results from the initial 16-week treatment period of the 52-week, 289 patient, Phase III ECZTRA 6 trial, where 21.4% of patients treated with tralokinumab 150 mg and 17.5% that received 300 mg achieved an IGA score of 0 or 1 compared to 4.3% with placebo in adolescents with moderate-to-severe atopic dermatitis who were candidates for systemic therapy. EASI-75 was achieved by 28.6% of patients who received tralokinumab 150 mg and 27.8% with 300 mg compared to 6.4% with placebo. Erytech announced that in the 512 patient, Phase III, TRYbeCA-1 trial, adding eryaspase to gemcitabine and nabpaclitaxel did not improve overall survival compared to gemcitabine plus nabpaclitaxel alone in patients with metastatic pancreatic cancer Published Research Updates In the 52-week, 2,002 patient, open-label, Phase III, SURPASS-4 trial, treatment with tirzepatide reduced HbA1c by 2.43% with 10 mg and 2.58% with 15 mg compared to a 1.44% decrease with insulin glargine in type 2 diabetics, inadequately controlled with oral drugs, who were at increased risk for cardiovascular disease. 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. Regulatory Update
The FDA approved avacopan (Tavenos, ChemoCentryx), on 10/8/2021, as adjunctive treatment of severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis (granulomatosis with polyangiitis and microscopic polyangiitis), in combination with standard therapy. The FDA approved of allogeneic processed thymus tissue (Rethymic, Enzyvant), on 10/8/2021, as a one-time treatment of congenital athymia. An FDA review found maribavir to be superior to investigator-assigned treatment (IAT) (ganciclovir, valganciclovir, foscarnet, or cidofovir) in clearing CMV DNA from plasma in a population of transplant patients with refractory CMV. However, the FDA felt this was primarily due to discontinuations in the IAT group.
The FDA granted an Orphan Drug Designation to posoleucel for the treatment of virus-associated hemorrhagic cystitis. The FDA designated gantenerumab a Breakthrough Therapy for the treatment of Alzheimer’s disease. The WHO recommends the use of the GSK malaria vaccine among children in sub-Saharan Africa and other areas heavily impacted by the disease. The FDA designated tezepelumab an Orphan Drug for the treatment of eosinophilic esophagitis. Announced Research Updates Brickell announced that in the six-week, 350 patient, Phase III, Cardigan I trial, 49.3% of patients treated with sofpironium improved their Hyperhidrosis Disease Severity Measure-Axillary (HDSSM-Ax) score by at least 2 points and had a 129.5 mg decrease in their gravimetric sweat production compared to 29.4% and 99.3 mg with placebo in patients nine years of age and older with primary axillary hyperhidrosis. Brickell announced that in the six-week, 351 patient, Phase III, Cardigan II trial, 63.9% of patients treated with sofpironium improved their HDSSM-Ax score by at least 2 points and had a 145.9 mg decrease in their gravimetric sweat production compared to 47% and 131.7 mg with placebo in patients nine years of age and older with primary axillary hyperhidrosis. BMS announced that in the 12-week, 131 patient, Phase II, LATTICE-UC trial, treatment with deucravacitinib did not improve remission rate compared to placebo in patients with moderate to severe ulcerative colitis. Published Research Updates In a draft evidence report, ICER found the evidence of a health benefit for adding mavacamten to optimal treatment of heart failure promising but inconclusive compared to optimal therapy alone or adding disopyramide. Some concern was expressed for a reduction in left ventricular ejection fraction seen in some patients. Some analysts have forecast an annual cost for mavacamten of $75,000, but ICER estimated a health-benefit price benchmark (HBPB) of $12,000 to $15,000. In a 20 patient, Phase II trial, treatment with camrelizumab plus apatinib resulted in an objective response rate of 55% in patients with high-risk chemorefractory or relapsed gestational trophoblastic neoplasia. A 52-week, open-label extension of an unpublished 6-week, 117 patient, Phase III, blonanserin trial, found that 60 Japanese adolescents that completed the extension had a 24.9 point decrease in their PANSS score. In the 52-week, 247 patient, Phase IIb ARREST trial, treatment with aramchol did not decrease hepatic triglycerides by magnetic resonance spectroscopy compared to placebo in patients with NASH. In a secondary endpoint analysis, treatment with aramchol lead to resolution of NASH without a worsening of fibrosis compared to placebo (16.7% vs 5%). Regulatory Update
The FDA approved atogepant (Qulipta, AbbVie), on 9/29/2021, for the prevention of episodic migraine. The most common adverse events with atogepant are constipation, nausea and fatigue. The FDA approved maralixibat (Livmarli, Mirum Pharmaceuticals), on 9/29/2021, for the treatment of cholestatic pruritus in patients with Alagille syndrome. Maralixibat will only be available through the Mirum Access Plus (MAP) program, a single-source specialty pharmacy owned by Mirum Pharmaceuticals. Biogen and Eisai initiated a rolling BLA submission for lecanemab for the treatment of early Alzheimer's disease. The FDA accepted the NDA for 177Lu-PSMA-617 for the treatment of metastatic castration-resistant prostate cancer, which suggested a PDUFA date of 3/29/2022. Announced Research Updates Pfizer is evaluating fordadistrogene movaparvovec as treatment for Duchenne muscular dystrophy in a 99 patient, Phase III trial. Due to three cases of muscle weakness and heart inflammation in two of the cases, Pfizer is amending the Phase III trial protocol to exclude patients with mutations affecting exons 9 through 13, or a deletion that affects both exon 29 and exon 30. BE BRIGHT is a long-term extension of the BE READY, BE VIVID and BE SURE trials. UCB announced that in the 1,355 patient, open-label, Phase III, BE BRIGHT trial, treatment with bimekizumab resulted in PASI 90 being achieved by 91% of BE SURE patients who had originally received bimekizumab and 72.3% achieving PASI 100 at 104 weeks. Similar response rates were achieved by patients switched from adalimumab in BE SURE to bimekizumab in BE BRIGHT. 69.9% of BE VIVID patients switched from ustekinumab to bimekizumab achieved PASI 100 at week 100. Lilly announced that in the 52-week, 243 patient, SURPASS-3 CGM sub-study, 72.6% of patients treated with tirzepatide maintained blood glucose in the target range of 71-140 mg/dL compared to 48% of patients treated with insulin degludec. Tirzepatide patients also spent less time in the hypoglycemia range of 70 mg/dL or less (0.6% vs 1.0%). Lilly announced that in a 104-week, Phase II trial, 186 patients with moderate-to-severe active ulcerative colitis (UC) received mirikizumab for 12-weeks. 93 patients responded and were randomized to mirikizumab every 4 weeks or every 12 weeks. After 52-weeks, 92.5% (86/93) continued taking mirikizumab and 82.1% achieved rectal bleeding remission and 84.6% achieved stool frequency remission. After two years, 83.9% remained on mirikizumab with 85.9% maintaining rectal bleeding remission and 84.6% maintaining stool frequency remission. Published Research Updates In the 69 patient, Phase IlI, FOCUS4-C trial, treatment with adavosertib improved progression free survival compared to active monitoring (3.61 vs 1.87 months) in metastatic colorectal cancer patients with TP53 and RAS mutations. A 143 patient, Phase II trial was stopped early when an interim analysis of the first 123 patients found no improvement in overall survival when adding veliparib to mFOLFIRI compared to FOLFIRI alone (5.4 vs 6.5 months) in patients with metastatic pancreatic cancer. |
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