FDA Approvals, Highlights, and Summaries: Endocrinology

February 17, 2022

Nexviazyme (avalglucosidase alfa)

Avalglucosidase is a recombinant acid alpha-glucosidase (GAA) indicated for late-onset Pompe disease in patients aged 1 year and older. GAA is deficient in Pompe disease, which leads to a buildup of glycogen in skeletal and cardiac muscle cells, causing weakness and premature death from respiratory failure or heart failure.

Approval was based on the COMET phase 3 trial that compared avalglucosidase alfa (n = 51) with alglucosidase alfa (n = 49). Clinically meaningful improvement with avalglucosidase-alfa therapy over alglucosidase alfa was observed in respiratory function, ambulation, and functional endurance. Lancet Neurol. 2021 Dec;20(12):1012-1026

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 mellitus 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

Recorlev (levoketoconazole)

Levoketoconazole is the purified 2S,4R enantiomer of ketoconazole. Levoketoconazole inhibits key steps in cortisol and testosterone synthesis. It is indicated for treatment of endogenous hypercortisolemia in adults with Cushing syndrome for whom surgery is not an option or has not been curative.

Results from the open-label phase 3 SONICS trial and the phase 3 LOGICS double-blind, randomized, controlled trial showed levoketoconazole led to sustained normalization of mean 24-hour urinary free cortisol (mUFC) and improvement in glycemic control that was more pronounced in patients with diabetes mellitus. Front Endocrinol 2021 Apr 7;12 and J Endocr Soc 2021 May 3; 5(Suppl 1): A526

Zegalogue (dasiglucagon)

Dasiglucagon is a ready-to-use, stable glucagon analog indicated for severe hypoglycemia in pediatric and adult patients with diabetes mellitus. It is a glucagon receptor agonist that increases blood glucose concentration by activating hepatic glucagon receptors, thereby stimulating glycogen breakdown and release of glucose from the liver.

Other endocrinology approval

Wegovy (semaglutide) - New distinct brand and indication as an adjunct to a reduced-calorie diet and increased physical activity for long-term weight management in adults who are obese (body mass index [BMI] ≥30 kg/m2) or overweight (BMI 27 kg/m2) with at least 1 weight-related comorbid condition (eg, hypertension, type 2 diabetes mellitus, dyslipidemia).

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