FDA approves treatment for weight management in patients with Bardet-Biedl Syndrome aged 6 or older

Action

The U.S. Food and Drug Administration has approved a supplemental indication for Imcivree (setmelanotide) injection for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to Bardet-Biedl Syndrome (BBS). Imcivree is the first drug approved specifically for chronic weight management in patients with BBS, a rare genetic disorder associated with early-onset childhood obesity.

FDA approved Imcivree in November 2020 for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to the genetic conditions proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency, which cause severe obesity that begins at an early age.

Disease or Condition

BBS is a rare genetic disorder with highly variable symptoms which may include obesity, retinal degeneration, reduced kidney function, or extra digits of the hands or feet, among others.

Obesity for patients with BBS starts at an early age (typically by age 1) and is progressive. Available weight management options are generally unsuccessful in patients with obesity due to BBS.

Effectiveness

The safety and effectiveness of Imcivree was evaluated in a 66-week trial that enrolled 44 patients with a clinical diagnosis of BBS and obesity (body mass index [BMI] greater than or equal to 30 kg/m2 or BMI greater than or equal to the 97th percentile for pediatric patients), ages 6 years and older. The trial included an initial 14-week, randomized, double-blind, placebo-controlled treatment period followed by a 52-week, open-label period. After 52 weeks of treatment, patients taking Imcivree lost, on average, 7.9% of their BMI. Sixty-one percent (61%) of patients lost 5% or more of their BMI, and 39% lost 10% or more of their BMI.

Safety Information

Imcivree comes with warnings and precautions: Imcivree is associated with disturbance in sexual arousal; male patients who have an erection lasting longer than 4 hours are instructed to seek emergency medical attention; Imcivree is associated with suicidal ideation and depression; therefore, individuals should be monitored for mood changes and suicidal thoughts; Imcivree is also associated with increases in skin pigmentation, including changes to pre-existing moles; therefore, individuals should undergo a skin examination before and during setmelanotide treatment. The most common adverse reactions include skin hyperpigmentation; injection site reactions; gastrointestinal reactions (nausea, vomiting, diarrhea); and spontaneous penile erection.

Designations

Imcivree received priority review, orphan drug designation, and breakthrough designation for this indication.

FDA granted this supplement approval to Rhythm Pharmaceuticals, Inc.