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Understanding Generalized Lipodystrophy (GL)

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Recognize and Diagnose GL

Patients may present with:

  • Reduced or complete lack of subcutaneous adipose tissue1
  • Elevated HbA1c levels, requiring extremely high doses of insulin to attain glycemic control2,3
  • Elevated triglyceride (TG) levels refractory to standard treatments1,3

Patients may report3,4:

  • Pain
  • Fatigue
  • Body image issues
  • Excessive hunger

There Are 2 Distinct Types of GL That Present Differently

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1

Congenital GL

  • Typically presents as generalized lack of subcutaneous fat in infancy to early childhood1
  • Autosomal recessive5
  • Negative genetic testing does not rule out GL (since not all mutations have been identified)3
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2

Acquired GL5,6

  • Can present as panniculitis or generalized subcutaneous fat loss in childhood to adulthood
  • More common in females
  • Autoimmune-related or idiopathic origin
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TIMING OF GL
Symptoms may present from birth to early childhood, or later in life. If there is widespread lack or loss of adipose tissue, consider GL in the differential diagnosis.1

GL Is a Disease of Leptin Deficiency

GL is a rare and heterogeneous disorder characterized by the loss of adipose tissue. Lack of adipose tissue can result in low levels of circulating leptin.1,6

Leptin is a hormone predominantly secreted by adipose tissue. It is responsible for regulating key metabolic functions7

  • Leptin informs the central nervous system of the status of energy stores in the body7
  • Leptin deficiency contributes to:

    • Insulin resistance1
    • Hypertriglyceridemia1
    • Diabetes mellitus1
    • Hepatic steatosis (fatty liver)1
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COULD IT BE GL?
These metabolic abnormalities are typically more severe in patients with GL and are often refractory to conventional lipid- and glucose-lowering agents.3

Only Myalept Addresses the Underlying Leptin Deficiency in GL

See How
References: 1. Cleveland Clinic. Lipodystrophy: what it is, symptoms, types & treatment. Cleveland Clinic. Accessed January 27, 2025, from https://my.clevelandclinic.org/health/diseases/23441-lipodystrophy 2. Marroquí L, Gonzalez A, Ñeco P, et al. Role of leptin in the pancreatic β-cell: effects and signaling pathways. J Mol Endocrinol. 2012;49(1):R9-R17. doi:10.1530/JME-12-0025 3. Akinci B, Gular MC, Oral EA. Lipodystrophy Syndromes: Presentation and Treatment. In: Feingold KR, Anawalt B, Blackman MR, et al, eds. Endotext [Internet]. MDText.com, Inc. Updated August 21, 2024. Accessed January 28, 2025. https://www.ncbi.nlm.nih.gov/books/NBK513130/ 4. Cook K, Adamski K, Gomes A, et al. Effects of metreleptin on patient outcomes and quality of life in generalized and partial lipodystrophy. J Endocr Soc. 2021;5(4):1-16. 5. Patni N, Garg A. Lipodystrophy for the diabetologist—what to look for. Curr Diab Rep. 2022;22(9):461-470. doi:10.1007/s11892-022-01485-w 6. Handelsman Y, Oral EA, Bloomgarden ZT, et al. The clinical approach to the detection of lipodystrophy—an AACE consensus statement. Endocr Pract. 2013;19(1): 107-116. 7. Picó C, Palou M, Pomar CA, Rodríguez AM, Palou A. Leptin as a key regulator of the adipose organ. Rev Endocr Metab Disord. 2022;23(1):13-30. doi:10.1007/s11154-021-09687-5

Important Safety Information

INDICATION: Myalept® (metreleptin) for injection is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.

LIMITATIONS OF USE: The safety and effectiveness of Myalept for the treatment of complications of partial lipodystrophy or for the treatment of liver disease, including nonalcoholic steatohepatitis (NASH), have not been established.

Myalept is not indicated for use in patients with HIV-related lipodystrophy. Myalept is not indicated for use in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy.

WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA

Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with Myalept. The consequences of these neutralizing antibodies are not well characterized but could include inhibition of endogenous leptin action and/or loss of Myalept efficacy. Severe infection and/or worsening metabolic control have been reported. Test for anti-metreleptin antibodies with neutralizing activity in patients who develop severe infections or show signs suspicious for loss of Myalept efficacy during treatment. Contact Chiesi Farmaceutici S.p.A. at 1-866-216-1526 for neutralizing antibody testing of clinical samples.

T-cell lymphoma has been reported in patients with acquired generalized lipodystrophy, both treated and not treated with Myalept. Carefully consider the benefits and risks of treatment with Myalept in patients with significant hematologic abnormalities and/or acquired generalized lipodystrophy.

Because of these risks associated with the development of anti-metreleptin antibodies that neutralize endogenous leptin and/or Myalept and the risk for lymphoma, Myalept is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myalept REMS PROGRAM.

CONTRAINDICATIONS: Myalept is contraindicated in general obesity not associated with congenital leptin deficiency. Myalept has not been shown to be effective in treating general obesity. The development of anti-metreleptin neutralizing antibodies have been reported in obese patients treated with Myalept. Myalept is contraindicated in patients with prior severe hypersensitivity reactions to metreleptin or to any of its components.

WARNINGS AND PRECAUTIONS: A dose adjustment, including possible large reductions, of insulin or insulin secretagogue may be necessary in some patients to minimize risk of hypoglycemia. Closely monitor blood glucose in patients on concomitant insulin, especially those on high doses, or insulin secretagogue.

Cases of progression of autoimmune hepatitis and membranoproliferative glomerulonephritis (associated with massive proteinuria and renal failure) were observed in some patients with acquired generalized lipodystrophy treated with Myalept. A causal relationship between Myalept and the development and/or progression of autoimmune disease has not been established. Carefully consider the benefits and risks of Myalept treatment in patients with autoimmune disease.

Hypersensitivity reactions (eg, anaphylaxis, urticaria or generalized rash) have been reported. Patient should promptly seek medical advice about discontinuation of Myalept if a hypersensitivity reaction occurs.

Myalept contains benzyl alcohol when reconstituted with Bacteriostatic Water for Injection. The preservative benzyl alcohol has been associated with serious adverse events and death in pediatric patients, particularly in neonates and premature infants. Preservative-free Water for Injection is recommended for use in neonates and infants.

ADVERSE REACTIONS: Most common adverse reactions (≥10%) in clinical trials were headache, hypoglycemia, decreased weight, and abdominal pain.

Please see Full Prescribing Information, including Boxed Warning.

Important Safety Information

INDICATION: Myalept® (metreleptin) for injection is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.

LIMITATIONS OF USE: The safety and effectiveness of Myalept for the treatment of complications of partial lipodystrophy or for the treatment of liver disease, including nonalcoholic steatohepatitis (NASH), have not been established.

Myalept is not indicated for use in patients with HIV-related lipodystrophy. Myalept is not indicated for use in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy.

WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA

Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with Myalept. The consequences of these neutralizing antibodies are not well characterized but could include inhibition of endogenous leptin action and/or loss of Myalept efficacy. Severe infection and/or worsening metabolic control have been reported. Test for anti-metreleptin antibodies with neutralizing activity in patients who develop severe infections or show signs suspicious for loss of Myalept efficacy during treatment. Contact Chiesi Farmaceutici S.p.A. at 1-866-216-1526 for neutralizing antibody testing of clinical samples.

T-cell lymphoma has been reported in patients with acquired generalized lipodystrophy, both treated and not treated with Myalept. Carefully consider the benefits and risks of treatment with Myalept in patients with significant hematologic abnormalities and/or acquired generalized lipodystrophy.

Because of these risks associated with the development of anti-metreleptin antibodies that neutralize endogenous leptin and/or Myalept and the risk for lymphoma, Myalept is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myalept REMS PROGRAM.

CONTRAINDICATIONS: Myalept is contraindicated in general obesity not associated with congenital leptin deficiency. Myalept has not been shown to be effective in treating general obesity. The development of anti-metreleptin neutralizing antibodies have been reported in obese patients treated with Myalept. Myalept is contraindicated in patients with prior severe hypersensitivity reactions to metreleptin or to any of its components.

WARNINGS AND PRECAUTIONS: A dose adjustment, including possible large reductions, of insulin or insulin secretagogue may be necessary in some patients to minimize risk of hypoglycemia. Closely monitor blood glucose in patients on concomitant insulin, especially those on high doses, or insulin secretagogue.

Cases of progression of autoimmune hepatitis and membranoproliferative glomerulonephritis (associated with massive proteinuria and renal failure) were observed in some patients with acquired generalized lipodystrophy treated with Myalept. A causal relationship between Myalept and the development and/or progression of autoimmune disease has not been established. Carefully consider the benefits and risks of Myalept treatment in patients with autoimmune disease.

Hypersensitivity reactions (eg, anaphylaxis, urticaria or generalized rash) have been reported. Patient should promptly seek medical advice about discontinuation of Myalept if a hypersensitivity reaction occurs.

Myalept contains benzyl alcohol when reconstituted with Bacteriostatic Water for Injection. The preservative benzyl alcohol has been associated with serious adverse events and death in pediatric patients, particularly in neonates and premature infants. Preservative-free Water for Injection is recommended for use in neonates and infants.

ADVERSE REACTIONS: Most common adverse reactions (≥10%) in clinical trials were headache, hypoglycemia, decreased weight, and abdominal pain.

Please see Full Prescribing Information, including Boxed Warning.