AZ’s orphan drug Myalept approved

AstraZeneca’s orphan drug Myalept has been approved in the US for patients with lipodystrophy.

Myalept (metreleptin for injection) is indicated as an adjunct to diet as replacement therapy for the treatment of complications of leptin deficiency in patients with congenital or acquired generalised lipodystrophy. The drug is a recombinant analogue of human leptin, and is the first treatment approved by the FDA for these patients.

AstraZeneca is in the process of taking over the rights to the drug from Bristol-Myers Squibb Company as part of the acquisition of the diabetes alliance assets, which was completed on 1 February.

Lipodystrophy is a group of rare syndromes which causes widespread loss of fat tissue under the skin. This in turn causes a deficit in the hormone leptin, leading to multiple metabolic complications. In some patients it is genetic, while other patients can develop it for different pathophysiological, and sometimes unknown, reasons.

AstraZeneca pointed out that the safety and effectiveness of Myalept to treat complications of partial lipodystrophy or for the treatment of liver disease, including non-alcoholic steatohepatitis (NASH), have not been established. Myalept is also not indicated for use in patients with HIV-related lipodystrophy or for use in patients with metabolic disease, including diabetes mellitus and hypertriglyceridaemia, without concurrent evidence of congenital or acquired generalised lipodystrophy.

Myalept has boxed warnings relating to the risk of anti-metreleptin antibodies with neutralising activity and risk of lymphoma. Because of these risks, Myalept’s prescribing will be tightly controlled and be monitored via a Risk Evaluation and Mitigation Strategy (REMS).

Briggs Morrison, Executive Vice President, Global Medicines Development and Chief Medical Officer, AstraZeneca welcomed the approval, saying Myalept represented a significant advance for people living with the disorder. He added that AstraZeneca was committed to supporting this patient community and would ensure patients prescribed the drug would have comprehensive patient support programme available to them.


Rare diseases – where are the patients?



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