Drug updated on 4/16/2024
| Dosage Form | Injection (subcutaneous; sterile, white, solid, lyophilized cake of 11.3 mg metreleptin per vial to deliver 5 mg per mL when reconstituted in 2.2 mL of BWFI or WFI) |
| Drug Class | Leptin analogs |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Used as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
Summary
- Metreleptin (Myalept) is utilized as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. It is effective in lowering triglycerides and Haemoglobin A1c levels across various genetic subgroups, including those with mutations in LMNA, PPARG, AGPAT2, or BSCL2 genes.
- Two systematic reviews/meta-analyses provided a comprehensive comparison and detailed insights into the safety and effectiveness of metreleptin.
- Regarding Body Mass Index reduction, metreleptin showed promising results among patients with partial and generalized lipodystrophy and specific genetic subgroups such as LMNA or BSCL2.
- Compared to thiazolidinediones, which also improved metabolic markers among all lipodystrophy patients, Recombinant human IGF-1, either alone or combined with IGFBP3, was effective specifically for the INSR-related insulin resistance subgroup.
- Monogenic Insulin Resistance (IR) patients were primarily focused on due to their significant influence on treatment outcomes. For the LEPT/LEPR Deficiency population, metreleptin has been effectively used, but there is a risk associated with the development of neutralizing antibodies, possibly leading to a decline in long-term efficacy for some individuals within this group.
- Despite its demonstrated benefits among certain populations suffering from complicated cases of lipodystrophy, caution must be exercised due to the low-to-very-low quality evidence available regarding its use along with potential risks such as the presence of neutralizing antibodies over long-term usage that warrant further research and close monitoring.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Myalept (metreleptin) prescribing information. | 2022 | Amryt Pharmaceuticals DAC, Dublin, Ireland |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Genotype-stratified treatment for monogenic insulin resistance: a systematic review. | 2023 | Communications Medicine |
| A national multicenter study of leptin and leptin receptor deficiency and systematic review. | 2023 | The Journal of Clinical Endocrinology and Metabolism |