Metreleptin
| Metreleptin | ||
|---|---|---|
| other names |
|
|
| Mass / length primary structure | 16,156 g · mol -1 | |
| Identifier | ||
| External IDs |
|
|
| Drug information | ||
| ATC code | A16 AA07 | |
| DrugBank | DB09046 | |
Metreleptin is a drug used in leptin replacement therapy. The protein metreleptin is a recombinant analogue of human ( i.e. human) leptin. Metreleptin is used to treat the very rare orphan disease lipodystrophy .
In the USA, metreleptin has been approved by the American regulatory authority (FDA) under the trade name Myalept since 2014 . For the EU, it was approved as Myalepta in July 2018 .
properties
Metreleptin is produced as a recombinant protein in E. coli . The metreleptin is then purified . Metreleptin consists of 147 amino acids with a disulfide bridge between cysteines at positions 97 and 147.
Clinical information
application areas
Metreleptin is a recombinant human leptin analog intended as a supplement to a diet in replacement therapy for the treatment of complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. It is injected subcutaneously .
Lipodystrophy is very rare , with a prevalence of around 1 in 1 million. Metreleptin was designated an orphan medicinal product for the EU in July 2012 .
Contraindications and other restrictions on use
Metreleptin must not be used in people who are generally overweight and are not associated with congenital lipodystrophy, or if they are hypersensitive to metreleptin.
The safety and efficacy of metreleptin in the treatment of partial lipodystrophy or in the treatment of liver disease, including steatohepatitis , have not been adequately studied. Metreleptin is not intended for use in patients with HIV-associated lipodystrophy as a result of antiretroviral therapy , or in patients with any metabolic disease, including diabetes mellitus and hypertriglyceridemia .
unwanted effects
Anti-metreleptin antibodies with neutralizing properties were observed as side effects .
Studies
About 30 studies are currently running, which are listed at clinicaltrials.gov.
Trade names
The trade name for metreleptin in the EU is Myalepta ; The manufacturer is Aegerion (EU). The trade name in the US is Myalept ; The manufacturer is Novelion . Aegerion is the European subsidiary of Novelion.
literature
- JCEM: Brown Rebecc J, et al .: The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline . In: The Journal of Clinical Endocrinology & Metabolism . December 2016, doi : 10.1210 / jc.2016-2466 .
Individual evidence
- ↑ a b Myalepta-Metreleptin , EPAR of the EMA (English), accessed on November 15, 2018
- ↑ An overview of Myalepta and why it is authorized in the EU , EMA, accessed on November 15, 2018
- ↑ Drugs @ FDA: FDA Approved Drug Products , FDA: Approval Date (s) and History, Letters, Labels, Reviews for BLA 125390, accessed September 4, 2017.
- ↑ Novelion Therapeutics Announces Marketing Authorization for MYALEPTA® (metreleptin) in the European Union to Treat Generalized and Partial Lipodystrophy , PM Novelion of December 21, 2016, accessed on November 15, 2018.
- ↑ a b c d e f g h Novelion Therapeutics' Subsidiary Files for European Approval for Metreleptin as a treatment for Generalized Lipodystrophy and a Subset of Patients with Partial Lipodystrophy , PM Novelion of December 21, 2016, accessed on November 15, 2018.
- ↑ a b DailyMed - MYALEPT - metreleptin injection, powder, lyophilized, for solution. In: dailymed.nlm.nih.gov. September 1, 2015, accessed September 20, 2017 .
- ↑ a b c d e f How Myalept works , product website (English).
- ↑ Lipodystrophy - Adipose tissue deficiency with fatal consequences , PZ Pharmazeutische Zeitung edition 24/2017, accessed on September 4, 2017.
- ↑ Overview of the currently running studies at clinicaltrials.gov
