FDA Approvals, Highlights, and Summaries: Nephrology and Urology

February 17, 2022

Korsuva (difelikefalin)

Difelikefalin is a peripherally restricted agonist of kappa opioid receptors thought to be essential in modulating pruritus. The drug is indicated for moderate-to-severe pruritus associated with chronic kidney disease in adults undergoing hemodialysis.

Results from the phase-3 KALM-1 trial showed 51.9% of patients in the difelikefalin group had at least a 3-point decrease in the 24-hour Worst Itching Intensity Numerical Rating Scale (WI-NRS), as compared to 30.9% in the placebo group. N Engl J Med. 2020 Jan 16;382(3):222-232

Kerendia (finerenone)

Finerenone is indicated to reduce risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, nonfatal myocardial infarction, and hospitalization for heart failure in adults with chronic kidney disease (CKD) associated with type 2 diabetes mellitus. It is the first nonsteroidal mineralocorticoid receptor (MR) antagonist to be approved for this purpose.

Approval was based on the FIDELIO-DKD trial, a placebo-controlled study that involved over 5700 patients with type 2 diabetes to whom the maximum-tolerated dose of renin-angiotensin system inhibitor (RASI) was already being administered. However, until more data on finerenone are gathered, RASIs and SGLT-2 inhibitors will be the preferred agents for slowing CKD in type 2 diabetes mellitus. N Engl J Med. 2020 Dec 3;383(23):2219-2229

Lupkynis (voclosporin)

Voclosporin is an oral calcineurin inhibitor indicated in combination with a background immunosuppressive regimen for treatment of active lupus nephritis in adults. 

The AURA-LV phase 3 trial randomized patients to placebo or an induction regimen that combined voclosporin with mycophenolate mofetil and low-dose oral corticosteroids. At 24 weeks, 32.6% of the low-dose voclosporin group achieved complete renal response (CRR), and 27.3% in the high-dose voclosporin group achieved CRR, as compared to 19.3% in the placebo group. CRR rate in the low-dose and high-dose voclosporin groups was also higher than that of placebo at 48 weeks. Kidney. Jan;95(1):219-231

The AURORA phase 3 trial compared the efficacy and safety of voclosporin (23.7 mg twice daily) with placebo in combination with mycophenolate and low-dose oral corticosteroids. At 1 year, the renal response rate was higher in the voclosporin group than in the placebo group (40.8% vs 22.5%; odds ratio, 2.65; P<.001). In addition, the median time to the achievement of a urine protein-to-creatinine ratio below 0.5 mg/mg showed significant clinical improvement with voclosporin when compared to placebo (169 vs 372 days; P<.001). Lancet. 2021 May 29;397(10289):2070-2080

Other nephrology and urology approvals

Tarpeyo (budesonide) - Delayed-release capsule indicated to reduce proteinuria (increased protein levels in the urine) in adults with primary immunoglobulin A (IgA) nephropathy at risk of rapid disease progression.

Entadfi (finasteride/tadalafil) - New fixed-dose combination indication for benign prostatic hypertrophy.    

Farxiga (dapagliflozin ) - Indication approved to reduce risk of sustained estimated glomerular filtration rate decline, end-stage kidney disease, cardiovascular death, and hospitalization for heart failure in adults with chronic kidney disease who are at risk of progression.

Myrbetriq (mirabegron) - FDA approval gained for neurogenic detrusor overactivity in children aged 3 years and older.

Botox (onabotulinumtoxinA) - New indication approved for neurogenic detrusor overactivity in children aged 5 years and older who have an inadequate response to or are intolerant of anticholinergic medications.

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