Metreleptin (Myalepta)

Key Messages

• Reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class.
• The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada’s federal, provincial, and territorial governments, with the exception of Quebec.
• This review assesses metreleptin (Myalepta), 3 mg, 5.8 mg, and 11.3 mg, powder for solution, subcutaneous injection.
• Indication: As an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy patients:
  • with confirmed congenital generalized lipodystrophy (Berardinelli-Seip syndrome) or acquired generalized lipodystrophy (Lawrence syndrome) in adults and children 2 years of age and above
  • with confirmed familial partial lipodystrophy (PL) or acquired PL (Barraquer-Simons syndrome), in adults and children 12 years of age and above with persistent significant metabolic disease for whom standard treatments have failed to achieve adequate metabolic control.