The study, titled "Rate of Diabetes Remission in Generalized Lipodystrophy Patients Treated with Metreleptin," was a post-hoc analysis of a prospective, open-label study of 53 patients conducted at the
In this post-hoc analysis, treatment with metreleptin allowed one-third (N=18) of patients to stop treatment with all antidiabetic medications, including insulin, for one year or more. The study also suggests that 17 percent of patients (N=9) achieved remission of diabetes.
Fifty-three patients with diabetes mellitus were assessed after one year of leptin replacement therapy for complete, partial or prolonged diabetes remission, defined as:
Among the 9 patients who achieved diabetes remission, 6 patients were on insulin and received an average dose of 440 units per day prior to metreleptin treatment.
Treatment-emergent adverse events occurring in greater than 10 percent of patients were: weight loss, abdominal pain, hypoglycemia, decreased appetite, and headache. Treatment-related serious adverse events occurred in 4 percent of patients.
"Severe insulin resistance adds significant complexity to controlling blood glucose among generalized lipodystrophy patients who have developed diabetes," said
The post-hoc analysis was completed by Brown and
GL is characterized by irreversible widespread loss of adipose tissue leading to low leptin levels. The absence of leptin results in the deposition of fat in the liver and muscle tissue and can lead to severe insulin resistance.
Lipodystrophy syndromes (LD) are ultra-rare disorders characterized by the irreversible loss of adipose tissue. In patients with lipodystrophy syndromes, levels of leptin are often very low. Leptin is a naturally occurring hormone produced in adipose tissue and is an important regulator of energy homoeostasis, fat and glucose metabolism, reproductive capacity, and other diverse physiological functions.
With GL, the loss of fat affects the whole body. With partial lipodystrophy, the loss of fat typically occurs in
the arms, legs, head and trunk regions, while accumulation of fat may occur in other areas of the body, including the neck, face and intra-abdominal regions. Metreleptin is only approved in the
MYALEPT® (metreleptin) for injection is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. LIMITATIONS OF USE: The safety and effectiveness of MYALEPT for the treatment of complications of partial lipodystrophy or for the treatment of liver disease, including nonalcoholic steatohepatitis (NASH), have not been established. MYALEPT is not indicated for use in patients with HIV-related lipodystrophy. MYALEPT is not indicated for patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy.
Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with MYALEPT. T-cell lymphoma has been reported in patients with acquired generalized lipodystrophy, both treated and not treated with MYALEPT.
MYALEPT is available only through a restricted program called the MYALEPT REMS PROGRAM.
For more detailed information, please see full Prescribing Information, including boxed warning, for MYALEPT.
The research described here is conducted in part by the Intramural Research Program of the
Amanda Murphy, Director, Investor Relations & Corporate Communications Novelion Therapeutics857-242-5024 firstname.lastname@example.org
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