title: Efficacy, patient-reported outcomes and safety profile of ATX-101 (deoxycholic acid), an injectable drug for the reduction of unwanted submental fat: results from a phase III, randomized, placebo-controlled study.
abstract: BACKGROUND
  Unwanted submental fat (SMF) may result in an unattractive chin profile and dissatisfaction with appearance. An approved and rigorously tested non-surgical method for SMF reduction is lacking.
  
  
  OBJECTIVE
  To evaluate the efficacy and safety of ATX-101 for the pharmacological reduction of unwanted SMF in a phase III randomized, double-blind, placebo-controlled study.
  
  
  METHODS
  Patients (n = 360) with moderate or severe SMF were randomized to receive ATX-101 1 or 2 mg/cm(2) or placebo injected into their SMF for up to four treatments ~28 days apart, with a 12-week follow-up. Coprimary efficacy endpoints were the proportions of treatment responders, defined as a ≥1-point reduction in SMF on the Clinician-Reported Submental Fat Rating Scale (CR-SMFRS), and those satisfied with their appearance in association with their face and chin after treatment on the Subject Self-Rating Scale (SSRS score ≥4). Secondary efficacy endpoints included a ≥1-point improvement in SMF on the Patient-Reported Submental Fat Rating Scale (PR-SMFRS) and changes in the Patient-Reported Submental Fat Impact Scale (PR-SMFIS). Additional patient-reported outcomes and changes in the Skin Laxity Rating Scale were recorded. Adverse events (AEs) and laboratory test results were monitored.
  
  
  RESULTS
  Compared with placebo, a greater proportion of patients treated with ATX-101 1 and 2 mg/cm(2) showed a ≥1-point improvement in CR-SMFRS (58.3% and 62.3%, respectively, vs. 34.5% with placebo; P < 0.001) and patient satisfaction (SSRS score ≥4) with the appearance of their face and chin (68.3% and 64.8%, respectively, vs. 29.3%; P < 0.001). Patient-reported secondary efficacy endpoints showed significant improvements in SMF severity (PR-SMFRS; P = 0.009 for ATX-101 1 mg/cm(2) , P < 0.001 for ATX-101 2 mg/cm(2) vs. placebo) and emotions and perceived self-image (PR-SMFIS; P < 0.001). No overall worsening of skin laxity was observed. AEs were mostly transient, mild to moderate in intensity and localized to the treatment area.
  
  
  CONCLUSIONS
  ATX-101 was effective and well tolerated, and may be an alternative to surgery for patients desiring improvement of their submental profile.
authors.affiliation: Clinic of Aesthetic Surgery IENA, Paris, France.
authors.forename: B
authors.initials: B
authors.lastname: Ascher
authors.forename: K
authors.initials: K
authors.lastname: Hoffmann
authors.forename: P
authors.initials: P
authors.lastname: Walker
authors.forename: S
authors.initials: S
authors.lastname: Lippert
authors.forename: U
authors.initials: U
authors.lastname: Wollina
authors.forename: B
authors.initials: B
authors.lastname: Havlickova
mesh_terms: D000273:Adipose Tissue / Q000187:drug effects*; D000328:Adult; D002680:Chin; D003840:Deoxycholic Acid / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology*; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D007279:Injections, Subcutaneous; D008297:Male; D010919:Placebos; D016896:Treatment Outcome
chemical_list: C000599116:ATX-101 compound; D010919:Placebos; D003840:Deoxycholic Acid