Metreleptin (Myalepta) - leptin deficiency in lipodystrophy View of Metreleptin (Myalepta) | Canadian Journal of Health TechnologiesReturn to Article DetailsMetreleptin (Myalepta)
Metreleptin (Myalepta) - Leptin deficiency in lipodystrophy View of Metreleptin (Myalepta) | Canadian Journal of Health TechnologiesReturn to Article DetailsMetreleptin (Myalepta)
Metreleptin (Myalepta) - to treat the complications of leptin deficiency in lipodystrophy (LD) patients 1 Published 12 June 2023 1 SMC2559 metreleptin powder for solution for injection (Myalepta®) Amryt Pharmaceuticals DAC 5 May 2023 The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for the above product using the ultra-orphan framework: Indication . After 12 months of treatment in a single-arm, open-label study, metreleptin significantly improved the co-primary outcomes of change from baseline in HbA1c and percent change in fasting triglycerides. The co-primary outcomes are surrogate outcomes of metabolic control and there are no data on the effect of metreleptin on clinically relevant longer term complications. There are no controlled data
Effects of Metreleptin in Patients with Generalized Lipodystrophy Before vs After the Onset of Severe Metabolic Disease. Leptin replacement therapy with metreleptin improves metabolic abnormalities in patients with generalized lipodystrophy (GLD). Determine how timing of metreleptin initiation in the clinical course of GLD affects long-term metabolic health. Retrospective analysis of patients ≥ 6 months old with congenital (n=47) or acquired (n=16) GLD treated with metreleptin at the National Institutes of Health since 2001. Least squares means (LSM) for HbA1c, insulin area under the curve (AUC) from oral glucose tolerance tests, triglycerides, urine protein excretion, platelets, transaminases, and aspartate aminotransferase (AST) to Platelet Ratio Index (APRI) for early and late
Metreleptin (Myalepta) for treatment of lipodystrophy Metreleptin (Myalepta®) for treatment of lipodystrophy | Report | National Health Care Institute Go to content You are here: Home Publications Metreleptin (Myalepta®) for treatment of lipodystrophy Search within English part of National Health Care Institute Search Metreleptin (Myalepta®) for treatment of lipodystrophyZorginstituut Nederland has completed its assessment whether metreleptin (Myalepta®) for treatment of lipodystrophy can be accepted into the Medicine Reimbursement System (GVS).Download "Metreleptin (Myalepta®) for treatment of lipodystrophy" PDF document | 73 pages | 1.5 MB Report | 21-10-2019 Registered indicationMetreleptin (Myalepta®) is a orphan drug used for: * the treatment of patients two years of age and older
Case report: clinical improvements observed in first off-label metreleptin treatment of a patient with atypical anorexia nervosa. Off-label metreleptin treatment resulted in cognitive, emotional and behavioral improvements of patients with anorexia nervosa, who presented with hypoleptinemia. We now report a case study of a 16-year-old female patient with atypical anorexia nervosa who was treated off-label with metreleptin for 11 days. She had lost 21 kg over 6 months. Her body mass index at referral for inpatient treatment was 20 kg/m, her serum leptin level was just within the normal range (2.4 ng/ml). Dosing resulted in prominent improvements of mood and weight phobia entailing a comparatively brief inpatient treatment. The observed improvements are similar to those observed in patients
Metreleptin for congenital leptin deficiency NHS England » Metreleptin for congenital leptin deficiency (all ages) Skip to main content Cookies on the NHS England websiteWe’ve put some small files called cookies on your device to make our site work.We’d also like to use analytics cookies. These send information about how our site is used to a service called Google Analytics. We use will commission metreleptin treatment for patients with congenital leptin deficiency in accordance with the criteria outlined in this document.In creating this policy NHS England has reviewed this clinical condition and the options for its treatment. It has considered the place of this treatment in current clinical practice, whether scientific research has shown the treatment to be of benefit to patients
Suggestive Evidence for an Antidepressant Effect of Metreleptin Treatment in Patients with Lipodystrophy. Lipodystrophy (LD) syndromes are rare heterogeneous disorders characterized by reduction or absence of subcutaneous fat, low or nondetectable leptin concentrations in blood and impaired hunger/satiety regulation. Metreleptin treatment reverses metabolic complications and improves eating behavior in LD. Because depression in anorexia nervosa (AN), which is also characterized by hypoleptinemia, improves substantially upon treatment with metreleptin, we hypothesized that metreleptin substitution may be associated with an antidepressant effect in patients with LD, too. In this ancillary study, 10 adult patients with LD were treated with metreleptin. To assess depressive symptoms, the self
Therapeutic indications and metabolic effects of metreleptin in patients with lipodystrophy syndromes: Real-life experience from a national reference network. To describe baseline characteristics and follow-up data in patients with lipodystrophy syndromes treated with metreleptin in a national reference network, in a real-life setting. Clinical and metabolic data from patients receiving metreleptin in France were retrospectively collected, at baseline, at 1 year and at the latest follow-up during treatment. Forty-seven patients with lipodystrophy including generalized lipodystrophy (GLD; n = 28) and partial lipodystrophy (PLD; n = 19) received metreleptin over the last decade. At baseline, the median (interquartile range [IQR]) patient age was 29.3 (16.6-47.6) years, body mass index
Endogenous Leptin Concentrations Poorly Predict Metreleptin Response in Patients With Partial Lipodystrophy. Leptin replacement with metreleptin improves glycemia and hypertriglyceridemia in severely hypoleptinemic patients with generalized lipodystrophy (GLD), but its effects are variable in partially leptin-deficient patients with partial lipodystrophy (PLD). Compare 3 leptin assays (Study I ); identify diagnostic performance of leptin assays to detect responders to metreleptin for each assay (Study II). Study I: cross-sectional analysis of average bias between leptin assays. Study II: retrospective analysis of diagnostic accuracy of potential leptin cut points to detect clinical responders to metreleptin. National Institutes of Health; University of Michigan. Study I: Metreleptin-naïve
Long-Term Effects of Metreleptin in Rabson-Mendenhall Syndrome on Glycemia, Growth, and Kidney Function. Rabson-Mendenhall syndrome (RMS) is caused by biallelic pathogenic variants in the insulin receptor gene (INSR) leading to insulin-resistant diabetes, microvascular complications, and growth hormone resistance with short stature. Small, uncontrolled studies suggest that 1-year treatment with recombinant leptin (metreleptin) improves glycemia in RMS. This study aimed to determine effects of long-term metreleptin in RMS on glycemia, anthropometrics, the growth hormone axis, and kidney function. We compared RMS patients during nonrandomized open-label treatment with metreleptin (≥ 0.15 mg/kg/day) vs no metreleptin over 90 months (5 subjects in both groups at different times, 4 only in metreleptin
Rapid amelioration of anorexia nervosa in a male adolescent during metreleptin treatment including recovery from hypogonadotropic hypogonadism. With this case report we support our medical hypothesis that metreleptin treatment ameliorates starvation related emotional, cognitive and behavioral symptomatology of anorexia nervosa (AN) and show for the first time strong effects in a male patient with AN. A 15.9 year old adolescent with severe AN of eight-month duration was treated off-label with metreleptin. Hyperactivity was assessed with accelerometry. Visual analogue scales (VAS), validated self- and clinician rating scales and lab results tracked changes from baseline to end of the 24-day dosing period and a five-month follow-up. Substantial improvements of mood and eating disorder related
Myalept (metreleptin for injection) - To treat the complications of leptin deficiency Drug Approval Package: Brand Name (Generic Name) NDA # Drug Approval Package Quick Links: Skip to main page content Skip to Search Skip to Topics Menu Skip to Common Links * * * U.S. Food & Drug Administration * Follow FDA * En EspañolSearch FDA * Home * Food * Drugs * Medical Devices * Radiation-Emitting Products * Vaccines, Blood & Biologics * Animal & Veterinary * Cosmetics * Tobacco ProductsDrug Approval Package * * * * FDA Home * Drugs * Drug Approvals and Databases * Drugs@FDA-Myalept (metreleptin)Company: Amylin Pharmaceuticals, LLCApplication No.: 125390Approval Date: 2/24/2014Persons with disabilities having problems accessing the PDF files below may call (301) 796-3634 for assistance
Metreleptin (metreleptin (genetical recombination)) This English version of the Japanese review report is intended to be a reference material to provide convenience for users. In the event of inconsistency between the Japanese original and this English translation, the former shall prevail. The PMDA will not be responsible for any consequence resulting from the use of this English version. Report on the Deliberation Results March 15, 2013 Evaluation and Licensing Division, Pharmaceutical and Food Safety Bureau Ministry of Health, Labour and Welfare [Brand name] Metreleptin for Subcutaneous Injection 11.25 mg “Shionogi” [Non-proprietary name] Metreleptin (Genetical Recombination) (JAN*) [Applicant] Shionogi & Co., Ltd. [Date of application] July 27, 2012 [Results
Efficacy of Metreleptin Treatment in Familial Partial Lipodystrophy Due to PPARG vs LMNA Pathogenic Variants. Familial partial lipodystrophy (FPLD) is most commonly caused by pathogenic variants in LMNA and PPARG. Leptin replacement with metreleptin has largely been studied in the LMNA group. To understand the efficacy of metreleptin in PPARG vs LMNA pathogenic variants and investigate predictors of metreleptin responsiveness. Subgroup analysis of a prospective open-label study of metreleptin in lipodystrophy. National Institutes of Health, Bethesda, Maryland. Patients with LMNA (n = 22) or PPARG pathogenic variants (n = 7), leptin <12 ng/mL, and diabetes, insulin resistance, or high triglycerides. Metreleptin (0.08 to 0.16 mg/kg) for 12 months. Hemoglobin A1c (HbA1c), lipids