Reduction of unwanted submental fat with ATX-101 (deoxycholic acid), an adipocytolytic injectable treatment: results from a phase III, randomized, placebo-controlled study

SUMMARY: BACKGROUND: Unwanted submental fat (SMF) is aesthetically unappealing, but methods of reduction are either invasive or lack evidence for their use. An injectable approach with ATX-101 (deoxycholic acid) is under investigation. OBJECTIVES: To evaluate the efficacy and safety of ATX-101 for the reduction of unwanted SMF. METHODS: In this double-blind, placebo-controlled, phase III study, 363 patients with moderate/severe SMF were randomized to receive ATX-101 (1 or 2 mg cm(−2)) or placebo injections into their SMF at up to four treatment sessions ∽28 days apart, with a 12-week follow-up. The co-primary efficacy endpoints were the proportions of treatment responders [patients with ≥ 1-point improvement in SMF on the 5-point Clinician-Reported Submental Fat Rating Scale (CR-SMFRS)] and patients satisfied with their face and chin appearance on the Subject Self-Rating Scale (SSRS). Secondary endpoints included skin laxity, calliper measurements and patient-reported outcomes. Adverse events were monitored. RESULTS: Significantly more ATX-101 recipients met the primary endpoint criteria vs. placebo: on the clinician scale, 59·2% and 65·3% of patients treated with ATX-101 1 and 2 mg cm(−2), respectively, were treatment responders vs. 23·0% for placebo (CR-SMFRS;P < 0·001); on the patient scale, 53·3% and 66·1%, respectively, vs. 28·7%, were satisfied with their face/chin appearance (SSRS;P < 0·001). Calliper measurements showed a significant reduction in SMF (P < 0·001), skin laxity was not worsened and patients reported improvements in the severity and psychological impact of SMF with ATX-101 vs. placebo. Most adverse events were transient and associated with the treatment area. CONCLUSIONS: ATX-101 was effective and well tolerated for nonsurgical SMF reduction. WHAT'S ALREADY KNOWN ABOUT THIS TOPIC? Unwanted submental fat (SMF) is considered aesthetically unappealing. Liposuction and face-lift are effective treatments for SMF reduction but are invasive and not suitable for all patients, whereas nonsurgical alternatives lack robust clinical evidence related to their safety and efficacy. ATX-101, a proprietary formulation of synthetically derived deoxycholic acid, is the first nonsurgical treatment for SMF reduction to be investigated in randomized, placebo-controlled clinical studies. WHAT DOES THIS STUDY ADD? This study provides the first data from a large-scale, randomized, placebo-controlled, phase III study of an injectable therapy for SMF reduction in a field currently lacking a sound evidence base. ATX-101 was superior to placebo for the clinician- and patient-evaluated reduction of unwanted SMF and led to improved patient perception about their appearance. ATX-101 was well tolerated; treatment-associated adverse events were mainly transient and localized injection-site reactions.