Drug updated on 9/4/2024
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.
Latest News
Summary
- Myalept (metreleptin) is used as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
- This summary is based on the review of two systematic review(s)/meta-analysis(es). [1-2]
- Metreleptin reduces triglycerides and HbA1c in all lipodystrophy cases (n = 111) and in LMNA, PPARG, AGPAT2, and BSCL2 subgroups (n = 72, 13, 21, and 21, respectively). It lowers BMI in partial/generalized lipodystrophy and in LMNA or BSCL2 subgroups but not in PPARG or AGPAT2 subgroups.
- Thiazolidinediones improve HbA1c and triglycerides in all lipodystrophy cases (n = 13) and show specific effectiveness in PPARG for HbA1c (n = 5) and LMNA for triglycerides (n = 7). Recombinant Human Insulin-like Growth Factor-1 (rhIGF-1) improves HbA1c in INSR-related insulin resistance (n = 17).
- Eleven LEP deficiency patients benefitted from long-term metreleptin treatment, with one patient developing neutralizing antibodies leading to a loss of efficacy.
- Safety outcomes beyond the development of neutralizing antibodies and hyperinsulinemia in LEP deficiency patients were not explicitly detailed in the reviewed studies.
- Metreleptin shows effectiveness in LMNA, PPARG, AGPAT2, and BSCL2 genetic subgroups and benefits patients with LEP deficiency, though there is a risk of developing neutralizing antibodies leading to loss of efficacy; LEP deficiency patients are diagnosed younger with higher BMI SD scores and higher rates of hyperinsulinemia compared to LEPR deficiency patients, who are associated with missense variants.
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 |