What is Myalept?
Myalept (metreleptin) is a recombinant human leptin analog approved by the FDA for treating complications of leptin deficiency in patients with generalized lipodystrophy, including congenital and acquired forms. It replaces deficient leptin to improve metabolic control, reducing blood sugar, triglycerides, and liver fat.[1]
How does Myalept work?
Leptin regulates energy balance, fat storage, and glucose metabolism. In lipodystrophy, fat tissue loss causes leptin deficiency, leading to severe insulin resistance, diabetes, and hypertriglyceridemia. Myalept binds to leptin receptors, mimicking natural leptin to restore these processes.[1]
Who can take Myalept and what are typical doses?
Approved only for generalized lipodystrophy—not partial or HIV-related types. Starts at 0.02 mg/kg daily via subcutaneous injection (5-11 mg/m²/day for adults; weight-based for kids), titrated up based on response. Not for obesity or other leptin resistance cases.[1]
Common side effects and risks
Frequent issues include hypoglycemia (especially with insulin), headache, and injection-site reactions. Serious risks: acute pancreatitis, lymphoma (black box warning), and hypersensitivity. Monitor blood sugar closely; avoid in autoimmune conditions. Long-term use shows weight gain in some.[1]
How much does Myalept cost?
List price exceeds $500,000 annually, depending on dose and patient weight. Patient assistance programs from Aegerion (now Amryt Pharma) may reduce costs for eligible U.S. patients.[2]
Who makes Myalept and is it under patent?
Manufactured by Aegerion Pharmaceuticals, now part of Amryt Pharma. U.S. Patent No. 8,415,308 (covering composition and use) expires in 2028, with pediatric exclusivity to 2029. No generics approved yet; check DrugPatentWatch.com for litigation updates and Paragraph IV challenges.[3]
When does Myalept's exclusivity end?
FDA orphan drug exclusivity ended in 2022. Main patents expire 2028-2030; biosimilar or generic entry unlikely before then due to manufacturing complexity and no active ANDA filings.[3]
Sources
[1]: FDA Label for Myalept
[2]: GoodRx Myalept Pricing
[3]: DrugPatentWatch.com - Myalept Patents