metreleptin

Metreleptin is a recombinant analogue of the human hormone leptin. It is used as replacement therapy for leptin deficiency in people with lipodystrophy, most notably congenital generalized lipodystrophy and generalized lipodystrophy with severe metabolic complications, and is sometimes used in selected forms of lipodystrophy with significant metabolic disease.

Mechanism and effects: Metreleptin acts on leptin receptors to restore leptin signaling, helping to improve insulin sensitivity and lipid metabolism. Clinically, treatment can reduce triglyceride levels and hepatic steatosis and may improve glycemic control and other metabolic abnormalities associated with lipodystrophy. Appetite modulation may also contribute to metabolic effects.

Administration and dosing: Metreleptin is given by subcutaneous injection, typically on a daily schedule. Dosing is individualized based on body weight and metabolic response, and therapy is generally continued long-term as long as benefits outweigh risks.

Regulatory status: In the United States, metreleptin was approved by the FDA for the treatment of leptin deficiency in patients with congenital generalized lipodystrophy or generalized lipodystrophy with severe metabolic complications; it is marketed as Myalept. In the European Union, it is marketed as Myalepta. Availability and indications vary by country.

Safety and monitoring: Common adverse events include injection-site reactions, headaches, and nausea. Some patients may develop anti-metreleptin antibodies, which can reduce efficacy. Rare immune-related adverse events have been reported. Patients receiving metreleptin require regular monitoring of metabolic parameters and immune status, and therapy should be managed by clinicians experienced with lipodystrophy.

See also: Leptin, lipodystrophy.