- |||||||||| Myalept (metreleptin) / Novelion
Journal: Editorial: Biological Therapies and Eating Disorders. (Pubmed Central) - Dec 16, 2024
Initiation date: Jul 2024 --> Jan 2025 Research of brain areas and bodily systems that are related to memory, emotions and immune function will hopefully expand the biological treatment options for people with EDs.
- |||||||||| Myalept (metreleptin) / Novelion
Budget Impact Analysis of Metreleptin in Patients With Lipodystrophy in Saudi Arabia () - Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_3039;
Research of brain areas and bodily systems that are related to memory, emotions and immune function will hopefully expand the biological treatment options for people with EDs. Given the uncertain assumptions regarding the total number of eligible patients and subgroups (GL and PL) for Metreleptin each year, small variations in patient numbers could have a significant effect on the overall budget impact.
- |||||||||| Myalept (metreleptin) / Novelion
Enrollment open: METRAN: Metreleptin in Anorexia Nervosa (clinicaltrials.gov) - Sep 19, 2024
P2, N=50, Recruiting,
Given the uncertain assumptions regarding the total number of eligible patients and subgroups (GL and PL) for Metreleptin each year, small variations in patient numbers could have a significant effect on the overall budget impact. Not yet recruiting --> Recruiting
- |||||||||| Myalept (metreleptin) / Novelion
Journal: Partial lipodystrophy: Clinical presentation and treatment. (Pubmed Central) - Jun 19, 2024
They are accompanied by multidisciplinary follow-up adapted to each clinical form. When standard treatments have failed to control metabolic disorders, the orphan drug metreleptin, an analog of leptin, can be effective in certain forms of lipodystrophy syndromes.
- |||||||||| Myalept (metreleptin) / Novelion
Journal: Leptin replacement therapy in the management of lipodystrophy syndromes. (Pubmed Central) - Jun 19, 2024
Metreleptin, a recombinant leptin analogue, was approved as an orphan drug to treat the metabolic complications of leptin deficiency in patients with generalized lipodystrophy in the USA or with either generalized or partial lipodystrophy in Japan and Europe. In this brief review, we will discuss the benefits and limitations of this therapy, and the new expectations arising from the recent development of a therapeutic monoclonal antibody able to activate the leptin receptor.
- |||||||||| Myalept (metreleptin) / Novelion
Enrollment closed, Phase classification, Trial completion date: Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL) (clinicaltrials.gov) - Apr 18, 2024
P2, N=4, Active, not recruiting,
Early treatment may also result is less severe progression of liver fibrosis, but further histological studies are needed to determine the effects of metreleptin therapy on liver disease. Enrolling by invitation --> Active, not recruiting | Phase classification: P1 --> P2 | Trial completion date: Jun 2024 --> Apr 2025
- |||||||||| Myalept (metreleptin) / Novelion
Review, Journal: Established and emerging treatments for eating disorders. (Pubmed Central) - Apr 15, 2024
In this review article, we first outline the evidence-base for the established treatments of the three 'classical' EDs [anorexia nervosa (AN), bulimia nervosa (BN), and binge eating disorder (BED)]. We then review research on some of the most promising emerging treatment modalities, discussing the questions and challenges that remain.
- |||||||||| Myalept (metreleptin) / Novelion
FAMILIAL PARTIAL LIPODYSTROPHY: CLINICAL FEATURES, GENETICS AND TREATMENT IN A GREEK REFERRAL CENTER () - Mar 17, 2024 - Abstract #ICEEDEC2024ICE_EDEC_1092;
We then review research on some of the most promising emerging treatment modalities, discussing the questions and challenges that remain. We have shown the presence of mutations both in exons, which are different from the ones already associated with the disease, and in introns, which might also contribute to the final amino acid products and the phenotype of the patient.The sustainable and favorable results of metreleptin treatment in FPLD patients were confirmed, and they are independent from any baseline parameters.
- |||||||||| Myalept (metreleptin) / Novelion
Enrollment closed: Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy (clinicaltrials.gov) - Mar 12, 2024
P4, N=10, Active, not recruiting,
We have shown the presence of mutations both in exons, which are different from the ones already associated with the disease, and in introns, which might also contribute to the final amino acid products and the phenotype of the patient.The sustainable and favorable results of metreleptin treatment in FPLD patients were confirmed, and they are independent from any baseline parameters. Recruiting --> Active, not recruiting
- |||||||||| Myalept (metreleptin) / Novelion
Journal: Classification of Congenital Leptin Deficiency. (Pubmed Central) - Mar 12, 2024
Recruiting --> Active, not recruiting Categorization of leptin variants based on molecular and functional characteristics helps to determine the most adequate approach to treatment of patients with congenital leptin deficiency.
- |||||||||| Myalept (metreleptin) / Novelion
Review, Journal: Does hypoleptinemia trigger entrapment in anorexia nervosa? Etiological and clinical considerations. (Pubmed Central) - Feb 2, 2024
We contrast the slow psychological recovery of patients with AN and of people who experienced starvation upon weight recovery with the rapid onset of improvements upon off-label metreleptin treatment. Characteristics of the sex and age dependent secretion of leptin may contribute to the elevated vulnerability of young females to develop AN.
- |||||||||| mibavademab (REGN4461) / Regeneron, Sanofi
Clinical, P1 data, Preclinical, Journal: Preclinical, randomized phase 1, and compassionate use evaluation of REGN4461, a leptin receptor agonist antibody for leptin deficiency. (Pubmed Central) - Nov 26, 2023
Furthermore, compassionate-use treatment of a single patient with atypical partial lipodystrophy and a history of undetectable leptin concentrations associated with neutralizing antibodies to metreleptin was associated with noteable improvements in circulating triglycerides and hepatic steatosis. Collectively, these translational data unveil an agonist LEPR mAb that may provide clinical benefit in disorders associated with relatively low leptin concentrations.
- |||||||||| Myalept (metreleptin) / Novelion, Imcivree (setmelanotide) / Rhythm Pharma
Review, Journal: Management of Monogenic and Syndromic Obesity. (Pubmed Central) - Nov 5, 2023
Collectively, these translational data unveil an agonist LEPR mAb that may provide clinical benefit in disorders associated with relatively low leptin concentrations. Additional therapies, including metreleptin and setmelanotide, that target defects within the leptin signaling pathway can effectively synergize with lifestyle efforts to treat monogenic disorders of leptin, leptin receptor, proopiomelanocortin (POMC), and proprotein convertase subtilisin/kexin type 1 (PCSK1) and syndromic conditions, such as the ciliopathies Bardet-Biedl and Alstr
- |||||||||| Myalept (metreleptin) / Novelion
Journal: Familial Partial Lipodystrophy: Clinical Features, Genetics and Treatment in a Greek Referral Center. (Pubmed Central) - Aug 16, 2023
In addition, treatment with metreleptin in specific FPLD patients significantly improved glycemic and lipidemic control, an effect which was sustained at the 12-month follow-up. More large-scale studies are necessary to clarify the genetic and allelic heterogeneity of the disease, along with other parameters which could predict treatment response.
- |||||||||| Myalept (metreleptin) / Novelion
Journal: Leptin decreases gluconeogenesis and gluconeogenic substrate availability in patients with lipodystrophy. (Pubmed Central) - Jul 29, 2023
Metreleptin treatment in patients with lipodystrophy reduced GNG likely through decreased availability of carbon sources for gluconeogenesis, such as alanine, lactate, and glycerol. Associations between alanine and GNG persisted after metreleptin treatment while correlations with glycerol and palmitate Ra did not persist, suggesting reduced importance of lipolysis as a driver of GNG in the leptin-replete state.
- |||||||||| Myalept (metreleptin) / Novelion
Journal: Metreleptin Robustly Increases Resting-state Brain Connectivity in Treatment-na (Pubmed Central) - Jul 5, 2023
Investigating brain connectivity alterations with metreleptin treatment using an independent sample of patients with LD, we have reproduced an increase of brain connectivity in hedonic and homeostatic central nervous networks observed previously with metreleptin treatment. These results are an important contribution to ascertain brain leptin action and help build a foundation for further research of central nervous effects of this important metabolic hormone.
- |||||||||| Myalept (metreleptin) / Novelion
Journal: The Metreleptin Effectiveness and Safety Registry (MEASuRE): concept, design and challenges. (Pubmed Central) - May 31, 2023
P=N/A
We propose that, through collaboration with existing registries and use of their established resources, patient enrolment timelines and data collection for new registries can be expedited. The learnings presented here may be applicable to other registries with similar objectives.
- |||||||||| Myalept (metreleptin) / Novelion
Leptin Decreases Gluconeogenesis and Gluconeogenic Substrate Availability in Patients with Lipodystrophy (W-178B) - May 11, 2023 - Abstract #ENDO2023ENDO_2318;
Reduced GNG may be mediated by improved insulin sensitivity leading to reductions in alanine and lactate, which are carbon sources for GNG. Associations between alanine and GNG persisted after metreleptin, suggesting that potential therapeutic interventions to reduce protein turnover in insulin-resistant states may improve glucose homeostasis.
- |||||||||| Myalept (metreleptin) / Novelion
Natural history of generalized lipodystrophy following early vs. late metreleptin treatment (ENDOExpo) - May 11, 2023 - Abstract #ENDO2023ENDO_1542;
Triglycerides were 96 mg/dL lower during follow-up between the early (<150 mg/dL) vs late (?150) groups (p=0.0116).In conclusion, patients with generalized lipodystrophy treated with metreleptin prior to the onset of clinically significant metabolic disease showed better age-adjusted metabolic control over time for diabetes, hypertriglyceridemia, and proteinuria. Lower insulin and C-peptide secretion in the late treatment group suggest that worse diabetes control after metreleptin in this group may be attributable to irreversible loss of beta cell function prior to metreleptin.
- |||||||||| Myalept (metreleptin) / Novelion
Effect of 48 Hour of Metreleptin in Humans With Lipodystrophy During Controlled Feeding (ENDOExpo) - May 11, 2023 - Abstract #ENDO2023ENDO_1213;
The long-term follow-up supports the safety profile of the treatment.*Unless otherwise noted, all abstracts presented at ENDO must not be released to the press or the public until the date and time of presentation. The effects of metreleptin on metabolism in LD appear to be rapid (48 hours), increase further after 12 days, and are independent from longer term changes in food intake, diabetes, and dyslipidemia.
- |||||||||| Myalept (metreleptin) / Novelion
Natural History of Pregnancy and Pregnancy Outcomes in Subjects with Lipodystrophy (ENDOExpo) - May 11, 2023 - Abstract #ENDO2023ENDO_1033;
6 (50%) had neonatal hypoglycemia.In summary, pregnancies in women with lipodystrophy showed high complication rates, including very high rates of miscarriage, diabetes, and pre-eclampsia. Larger studies are needed to determine how management of these patients during pregnancy, including with newer treatments such as metreleptin for GL, affects pregnancy and offspring outcomes.
- |||||||||| Vyvanse (lisdexamfetamine) / Shionogi, Takeda, Myalept (metreleptin) / Novelion
Novel psychopharmacological treatments for eating disorders (10. Room 152) - Mar 2, 2023 - Abstract #EPA2023EPA_104;
Case studies provide examples of successful use of RWE for HTA submission. The currently approved pharmacological treatments for EDs are limited to fluoxetine for bulimia nervosa (BN) and - in some countries
- |||||||||| Myalept (metreleptin) / Novelion
Enrollment open, Trial completion date, Trial initiation date, Trial primary completion date: Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL) (clinicaltrials.gov) - Feb 14, 2023
P1, N=2, Enrolling by invitation,
Eleven patients benefited from long-term metreleptin, with one losing efficacy due to neutralizing antibodies. Not yet recruiting --> Enrolling by invitation | Trial completion date: Oct 2022 --> Dec 2023 | Initiation date: Jul 2022 --> Mar 2023 | Trial primary completion date: Oct 2022 --> Dec 2023
- |||||||||| Myalept (metreleptin) / Novelion
Journal: Lipodystrophies in non-insulin-dependent children: treatment options and results from recombinant human leptin therapy. (Pubmed Central) - Dec 26, 2022
In this narrative review, we presentend the clinical presentation of different types of lipodystrophies and metabolic unbalances related to disease in children and adolescents, focusing on the main treatment options and the novel results from recombinant human leptin (metreleptin) therapy...New therapeutic strategies are currently being investigated, especially for patients with PL forms, specifically, liver-targeted therapies. Further studies are needed to achieve the most specific and precise treatment possible.
- |||||||||| Symlin (pramlintide) / AstraZeneca
Trial completion date, Trial primary completion date, MRI: A Study of the Functional Magnetic Resonance Imaging Response to Leptin and Pramlintide (clinicaltrials.gov) - Dec 16, 2022
P=N/A, N=10, Active, not recruiting,
Further studies are needed to achieve the most specific and precise treatment possible. Trial completion date: Jul 2022 --> Jul 2024 | Trial primary completion date: Jul 2022 --> Jul 2024
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