Bardet-Biedl syndrome (BBS) is a rare genetic disease1

Obesity and hyperphagia are highly prevalent in BBS1-3

  • Estimated prevalence (US): 1500-2500*
  • BBS is part of a class of disorders called ciliopathies (impairment of cilia function in cells)4
  • Cilia are hairlike projections that play a role in the regulation of several biological processes, including satiety signaling4
 
BBS is diagnosed clinically
 
 
Additional clinical features1,9
  • Speech delay
  • Developmental delay
  • Diabetes mellitus
  • Congenital heart disease
  • Dental anomalies
  • Brachydactyly/syndactyly
  • Ataxia/poor coordination
  • Anosmia/hyposmia

Consider the complete patient presentation and use your clinical judgment to diagnose BBS

*Estimated prevalence of US patients based on company estimates.

 

Hyperphagia is a hallmark of BBS that can intensify physical and mental challenges for patients and caregivers3,10

Hyperphagia often has an early onset, typically by age 53

  • 91% of caregivers of children with BBS reported increased interest in food before age 53
  • Patients with BBS who experience hyperphagia may:
    • Take a longer time to feel full while eating
    • Feel hungry again right after a meal

 

Dimensions of hyperphagia10

Hyperphagia Behavior
Behavior
Excessive food-seeking behavior
"I can’t stop myself from eating or sneaking food in the middle of the night."
Individual living with BBS11
Hyperphagia Drive
Drive
Preoccupation with food
"My daughter has told me, ‘It’s like I’m battling with my mind.’"
Caregiver of a child living with BBS11
Hyperphagia Severity
Severity
Impact on daily activities
"She is constantly hungry...24/7...She constantly cries for more food. I have to constantly tell her no... It’s very hard."
Caregiver of a child living with BBS11

 

 

Obesity is common in BBS and can seriously impact overall health1,5,7,12

9 out of 10 BBS patients
 

As many as 9 out of 10 patients with BBS are affected by obesity1

  • Severe obesity has an early onset, typically beginning in childhood by age 5, and persists into adulthood7
    • The risks associated with obesity accumulate over the lifespans of patients with BBS5,7
Obesity in patients with BBS
 

Obesity in patients with BBS can cause and/or accelerate comorbidities, including1,5,7,12-15

  • Diabetes
  • Renal impairment
  • Hypertension
 
 
References: 1. Forsythe E, Beales PL. Eur J Hum Genet. 2013;21(1):8-13. 2. Beales PL et al. J Med Genet. 1999;36(6):437-446. 3. Sherafat-Kazemzadeh R et al. Pediatr Obes. 2013;8(5):e64-e67. 4. Vaisse C et al. Cold Spring Harb Perspect Biol. 2017;9(7):a028217. 5. Forsythe E et al. Front Pediatr. 2018. doi:10.3389/fped.2018.00023. 6. Pigeyre M et al. Clin Sci (Lond). 2016;130(12):943-986. 7. Pomeroy J et al. Pediatr Obes. 2021;16(2):e12703. 8. Forsythe E et al. J Am Soc Nephrol. 2017;28(3):963-970. 9. Forsyth R, Gunay-Aygun M. GeneReviews®. July 14, 2003. Updated July 23, 2020. Accessed May 3, 2022. https://www.ncbi.nlm.nih.gov/books/NBK1363. 10. Heymsfield SB et al. Obesity (Silver Spring). 2014;22(01):S1-S17. 11. Data on file. Rhythm Pharmaceuticals, Inc. Boston, MA. 12. Forsythe E et al. Clin Genet. 2015;87(4):343-349. 13. Klein S et al. Cell Metab. 2022;34(1):11-20. 14. Kovesdy CP et al. Physiol Int. 2017;104(1):1-14. 15. Landsberg L et al. J Clin Hypertens (Greenwich). 2013;15(1):14-33.
Important Safety Information

WARNINGS AND PRECAUTIONS

Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur.

Skin Pigmentation and Darkening of Pre-existing Nevi: Generalized increased skin pigmentation and darkening of pre-existing nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmentary lesions.

Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and low birth weight infants treated with benzyl alcohol-preserved drugs.

ADVERSE REACTIONS

  • Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection

USE IN SPECIFIC POPULATIONS

Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at 833-789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see the full Prescribing Information for additional Important Safety Information.

Indication

IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to Bardet-Biedl syndrome (BBS).

Limitations of Use

IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

  • Other types of obesity not related to BBS or other FDA-approved indications for IMCIVREE, including obesity associated with other genetic syndromes and general (polygenic) obesity
Important Safety Information

WARNINGS AND PRECAUTIONS

Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.

Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur.

Skin Pigmentation and Darkening of Pre-existing Nevi: Generalized increased skin pigmentation and darkening of pre-existing nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmentary lesions.

Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and low birth weight infants treated with benzyl alcohol-preserved drugs.

ADVERSE REACTIONS

  • Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection

USE IN SPECIFIC POPULATIONS

Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.

To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at 833-789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see the full Prescribing Information for additional Important Safety Information.

Indication

IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to Bardet-Biedl syndrome (BBS).

Limitations of Use

IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:

  • Other types of obesity not related to BBS or other FDA-approved indications for IMCIVREE, including obesity associated with other genetic syndromes and general (polygenic) obesity