The study titled "Impact of Metreleptin on Hepatomegaly in Patients with Generalized Lipodystrophy" suggests treatment with metreleptin may reduce liver volume in patients with enlarged liver (hepatomegaly) resulting from complications of generalized lipodystrophy (GL). The study also demonstrated improvements from baseline of key metabolic parameters, including hemoglobin A1c (HbA1c), liver enzymes and triglycerides.
The post-hoc analysis of an open-label, prospective study in patients with GL assessed both the impact on liver volume and key metabolic parameters when patients were treated with metreleptin as leptin replacement therapy. Of the 34 evaluable patients, 22 had baseline liver volumetric measurements using MRI; all 22 patients had an enlarged liver. Normal liver volume was defined as less than 25 mL/kg of body weight.
The post-hoc analysis completed by
For patients assessed within one year of initiating treatment with metreleptin (N=21), liver volume decreased an average of 25 percent. The mean treatment duration was 10 months. Among these same patients who had additional follow ups after one year (N=14) and had a mean duration of metreleptin treatment of 46 months, liver volume decreased by 35 percent.
The most commonly reported adverse events occurring in more than 10 percent of patients were abdominal pain, hypoglycemia, ear infection, fatigue, proteinuria, back pain, diarrhea, headache, menorrhagia, nausea, ovarian cyst, upper respiratory tract infection and weight decline.
"GL patients lack fat cells and the absence of adipocytes can lead to the accumulation of fat in the liver that can potentially progress to various forms of liver disease," said
Patients treated with metreleptin also experienced clinically meaningful reductions from baseline in HbA1c, liver enzymes (both AST and ALT) and triglycerides, a type of fat in the blood.
Novelion believes that metreleptin may demonstrate clinical results in a wide range of low leptin-mediated rare and metabolic diseases. John Orloff, MD, executive vice president and head of research and development for Novelion, stated, "Analysis like this, combined with 14 years of additional patient data, provides evidence supporting the implications of low leptin levels on various physiologic functions. We are currently evaluating conducting clinical trials to investigate the use of metreleptin in multiple other low leptin-mediated conditions."
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 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 (
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. For more detailed information, please see additional Important Safety Information and the Prescribing Information, including boxed warning, for MYALEPT.
MYALEPT is available only through a restricted program called the MYALEPT REMS PROGRAM.
Certain statements in this press release constitute "forward-looking statements" of Novelion within the meaning of applicable laws and regulations and constitute "forward-looking information" within the meaning of applicable Canadian securities laws, including the statement regarding expectations as to label expansion for metreleptin. Forward-looking statements are based on estimates and assumptions made by Novelion in light of current conditions and expected future developments, as well as other factors that Novelion believes are appropriate in the circumstances, including but not limited to, our financial position and execution of
our business strategy, receipt of regulatory approvals, indication prevalence, and product competition, market acceptance, sales, pricing, reimbursement and side effects. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. Many such risks, uncertainties and other factors are taken into account as part of our assumptions underlying these forward-looking statements and include, among others, the following: the risks that metreleptin may not obtain regulatory approval for additional indications; that, if approved, metreleptin may not obtain reimbursement approvals on a timely basis or at all or at the levels necessary to support
commercialization; that the prevalence estimates for a desired indication may be inaccurate; the risk that orphan drug, data and marketing exclusivity or the patent protection for metreleptin may not be sufficient or may be invalidated; and the other risks common to seeking regulatory approval for, developing and commercializing orphan drugs. For additional disclosure regarding these and other risks we face, see the disclosure contained in the "Risk Factors" section of our Annual Report on Form 10-K filed on
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