Metreleptin (Myalepta)

Key Messages

• We recommend that Myalepta should be reimbursed by public drug plans as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in patients with lipodystrophy (LD) if certain conditions are met.
• Myalepta should be covered to treat patients with confirmed congenital generalized LD (GL) (Berardinelli-Seip syndrome) or acquired GL (Lawrence syndrome) in both adults and children who are at least 2 years old and have at least 1 metabolic abnormality of diabetes, insulin resistance, or high levels of triglycerides (TGs). Myalepta should also be covered to treat patients who have confirmed familial partial LD (PL) or acquired PL in adults and children who are at least 12 years old and have persistent significant metabolic abnormalities, even after trying other treatments for at least 12 months.
• Myalepta should only be reimbursed if prescribed by an endocrinologist or pediatric endocrinologist with expertise in treating LD and if the cost of Myalepta is reduced. When first prescribed, Myalepta should only be reimbursed for 12 months. Reimbursement may be renewed on a yearly basis for patients who show improvement in their blood sugar and/or triglyceride levels.