FDA

Imcivree now approved for younger children

December 31, 2024

Brand name: Imcivree

Generic name: setmelanotide

Manufacturer: Rhythm Pharmaceuticals

Approval date: December 20, 2024

FDA expanded the approval of Imcivree (setmelanotide) to include children ≥2 years of age with obesity due to Bardet-Biedl syndrome (BBS) or pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. Previously, it had been approved for patients ≥6 years of age.

Efficacy

Safety and efficacy of Imcivree for weight reduction in pediatric patients ages 2 to <6 years with obesity due to POMC, PCSK1, or LEPR deficiency or BBS were assessed in a 52-week VENTURE trial (NCT04966741). Patients with PCSK1 deficiency were eligible but none enrolled. POMC and LEPR deficiency were confirmed by genetic testing demonstrating biallelic variants interpreted as pathogenic, likely pathogenic, or of undetermined significance; BBS was diagnosed clinically with genetic confirmation. Obesity was defined as baseline BMI ≥97th percentile for age and sex and body weight ≥20 kg. Imcivree dose titration occurred over an 8-week period, followed by a 44-week open-label treatment period. Twelve patients were enrolled (3 patients with POMC deficiency, 4 patients with LEPR deficiency, and 5 patients with BBS); 58% were male; 58% were White, 8% were Asian, and 33% had an unknown or not reported race; 8% were Hispanic or Latino ethnicity and 17% had an unknown or not reported ethnicity; and mean BMI was 29.9 kg/m^2 (range: 19-43 kg/m^2) at baseline.

Mean percent change in BMI after 52 weeks of Imcivree treatment was -33.8%, -13.1%, and -9.7% in patients with POMC deficiency, LEPR deficiency, and BBS, respectively.

Safety

The most common adverse events reported in the trial were skin hyperpigmentation, injection site reactions, diarrhea, nausea, and headache.

Sources:

Rhythm Pharmaceuticals. (2024, December 20). Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients as Young as 2 Years Old. [Press release]. https://ir.rhythmtx.com/news-releases/news-release-details/rhythm-pharmaceuticals-announces-fda-approval-imcivreer-0

Rhythm Pharmaceuticals: Imcivree (setmelanotide). [Package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/213793s007lbl.pdf

Argente J, et al. (2025, January). Lancet Diabetes Endocrinol. Setmelanotide in patients aged 2-5 years with rare MC4R pathway-associated obesity (VENTURE): a 1 year, open-label, multicenter, phase 3 trial. https://pubmed.ncbi.nlm.nih.gov/39549719/