Metreleptin (Myalepta®). HTA ID: 25058

Assessment Status Rapid Review Complete
HTA ID 25058
Drug Metreleptin
Brand Myalepta®
Indication Metreleptin (Myalepta®) is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: • with confirmed congenital generalised LD (Berardinelli-Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children two years of age and above • with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.
Assessment Process
Rapid review commissioned 17/09/2025
Rapid review completed 13/10/2025
Rapid review outcome A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of metreleptin compared with the current standard of care.