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New HTA Decisions in England
March 2021
Drug name
MYALEPTA® (metreleptin)
Company
Amryt
Decision date
20/01/2021
Therapeutic area
Cardiovascular conditions
Therapeutic sub area
Lipid disorders
Decision
Recommended with restrictions
Indication
Metreleptin is recommended, within its marketing authorisation, as an option for treating the complications of leptin deficiency in lipodystrophy for people who are 2 years and over and have generalised lipodystrophy. Metreleptin is also recommended as an option for treating the complications of leptin deficiency in lipodystrophy for people who are 12 years and over, have partial lipodystrophy, and do not have adequate metabolic control despite having standard treatments. It is only recommended if they have an HbA1c level above 7.5%, or fasting triglycerides above 5.0 mmol/litre, or both.
Decision Detail
This is a highly specialised technologies guidance.
Summary
Clinical evidence submitted by the company included NIH 991265 (a pilot, dose-escalation study) with continued treatment in the extension study NIH 20010769 for long-term follow up, and FHA101 (an open-label, single-arm, expanded-access study). Evidence suggested metreleptin provides clinical benefits by reducing blood sugar, triglycerides and liver enzymes in people with lipodystrophy. The indirect treatment comparison results presented by the company during the resubmission also indicated that metreleptin was associated with greater improvement in metabolic outcomes compared with supportive care. However, metreleptin's long-term effect and several assumptions in the economic modelling are uncertain. The committee agreed that the ICER of £108,267 per QALY gained would lower to an acceptable range for metreleptin to be an effective use of NHS resources. Therefore, the committee concluded that metreleptin can be considered a cost-effective use of NHS resources for highly specialised technologies, and recommended metreleptin as an option for treating the complications of leptin deficiency in lipodystrophy for people who are either 2 years and over with generalised lipodystrophy, or 12 years and over with partial lipodystrophy with HbA1c above 7.5%, or fasting triglycerides above 5.0 mmol/litre, or both.