Rhythm Files for Imcivree Expanded Use

FDA has accepted for priority review a Rhythm Pharmaceuticals supplemental NDA for Imcivree (setmelanotide), a melanocortin-4 receptor agonist for treating obesity due to Bardet-Biedl syndrome or pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency in children as young as two years old. The agency has set a user fee review action target date of 12/26.

The submission is based on data from Rhythm’s multi-center, one-year, open-label Phase 3 trial in patients (N=12) between two years and six years of age in the targeted patient population. Rhythm says treatment with setmelanotide achieved the primary endpoint and demonstrated a 18.4% percent mean reduction in body mass index.

Currently, setmelanotide is FDA-approved for chronic weight management in adult and pediatric patients six years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency.