Novelion Therapeutics Inc. (NASDAQ:NVLN), a biopharmaceutical
company dedicated to developing new standards of care for
individuals living with rare diseases, today announced the
presentation of two separate studies by independent researchers at
the 10th International Meeting of Pediatric Endocrinology taking
place in Washington, D.C., September 14-17.
The first study was a cross-section analysis examining key
clinical characteristics of pediatric patients with confirmed
familial partial lipodystrophy (FPL). Of the 13 pediatric patients
18 years or younger:
- All had at least one metabolic complication resulting from FPL,
followed by diabetes (77 percent);
- The ages of onset were 10-18 years for NAFLD; 10-17 years for
hyperlipidemia; and 11-17 years for diabetes;
- Among those with diabetes, 85 percent required diabetes
medication and more than half were using insulin;
- Nearly one third of patients with hyperlipidemia were treated
with lipid-lowering agents; and
- Of the 12 patients who had liver imaging performed, 11 had
evidence of hepatosteatosis, or fatty liver.
The analysis was conducted in patients who were referred to the
National Institutes of Health between 2003-2017 and presented by
Rebecca Brown, M.D., Lasker Clinical Research Scholar, Section on
Translational Diabetes and Metabolic Syndromes, Diabetes,
Endocrinology, and Obesity Branch at the National Institutes of
Health.
“FPL is a rare disease, and there is significant clinical
variability among these patients, including differences in leptin
levels, percent body fat and triglycerides, which makes accurate
diagnosis of FPL challenging,” said Brown. “Early diagnosis of
pediatric FPL is important for management of these patients.”
A second study examined the effect of metreleptin on liver
volume among pediatric patients with generalized lipodystrophy
(GL). Of the 13 patients assessed, all had an enlarged liver
(hepatomegaly) at baseline.
For patients assessed within a year after initiating treatment
with metreleptin, liver volume decreased by 25 percent. The mean
duration of treatment was 9 months. Among patients who had longer
exposure to metreleptin (N=9) with a mean of 46 months, liver
volume decreased by 34 percent.
The most commonly reported adverse events included
gastrointestinal disorders, including abdominal pain and
pancreatitis, and the two patients who experienced pancreatitis had
reported episodes in their prior medical history.
This post-hoc analysis reviewed patients who participated in a
prospective, open-label study conducted by the National Institutes
of Health between 2000-2008.
“Fatty liver is a common complication among pediatric patients
with GL,” said Elif Oral, M.D., Associate Professor of Medicine,
Michigan Medicine. “Treatment with metreleptin was associated with
sustained reduction in liver volume during this study.”
About Lipodystrophy
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 generalized lipodystrophy, 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 U.S. to treat generalized lipodystrophy and is not
approved to treat partial lipodystrophy.
About MYALEPT® (metreleptin) for
injectionMYALEPT® (metreleptin) for injection is only
approved in the United States and 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. 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 use in
patients with metabolic disease, including diabetes mellitus and
hypertriglyceridemia, without concurrent evidence of generalized
lipodystrophy.
Highlights of Safety Information from U.S. Prescribing
Information
WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH
NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA
See full prescribing information for complete boxed
warning.
Anti-metreleptin antibodies with neutralizing activity
have been identified in patients treated with MYALEPT. The
consequences are not well characterized but could include
inhibition of endogenous leptin action and/or loss of MYALEPT
efficacy. Worsening metabolic control and/or severe infection have
been reported. Test for anti-metreleptin antibodies with
neutralizing activity in patients with severe infections or loss of
efficacy during MYALEPT treatment.
T-cell lymphoma has been reported in patients with
acquired generalized lipodystrophy, both treated and not treated
with MYALEPT. Carefully consider the benefits and risks of MYALEPT
treatment in patients with significant hematologic abnormalities
and/or acquired generalized lipodystrophy.
MYALEPT is available only through a restricted program
under a Risk Evaluation and Mitigation Strategy (REMS) called the
MYALEPT REMS PROGRAM.
CONTRAINDICATIONS
MYALEPT is contraindicated in general obesity not associated
with congenital leptin deficiency and in patients with
hypersensitivity to metreleptin.
WARNINGS AND PRECAUTIONS
Anti-metreleptin antibodies with neutralizing activity: Could
inhibit endogenous leptin action and/or result in loss of MYALEPT
efficacy. Test for neutralizing antibodies in patients with
severe infections or loss of efficacy during MYALEPT treatment.
T-cell lymphoma: Carefully consider benefits and risks of
treatment with MYALEPT in patients with significant hematologic
abnormalities and/or acquired generalized lipodystrophy.
Hypoglycemia: A dose adjustment, including possible large
reductions, of insulin or insulin secretagogue may be necessary.
Closely monitor blood glucose in patients on concomitant insulin,
or insulin secretagogue.
Autoimmunity: Autoimmune disorder progression has been
observed in patients treated with MYALEPT. Carefully consider
benefits and risks of MYLEPT treatment in patients with autoimmune
disease.
Hypersensitivity reactions (e.g., anaphylaxis, urticaria or
generalized rash) have been reported. Patients should
promptly seek medical advice about discontinuation of MYALEPT if a
hypersensitivity reaction occurs.
Benzyl Alcohol Toxicity: Preservative-free Water for
Injection is recommended for use in neonates and infants.
ADVERSE REACTIONS
Most common adverse reactions (≥10%) in clinical trials were
headache, hypoglycemia, decreased weight, and abdominal pain.
USE IN SPECIAL POPULATIONS
MYALEPT should be used during pregnancy only if the potential
benefit justifies the potential risk to the fetus. No
adequate and well-controlled studies have been conducted with
metreleptin in pregnant women. Nursing Mothers should discontinue
drug or nursing.
For additional information, please see the
U.S.
Prescribing Information including Box
Warning.
NIDDK DISCLAIMER
The research described here is conducted in part by the
Intramural Research Program of the National Institute of Diabetes
and Digestive and Kidney Diseases (NIDDK) of the National
Institutes of Health. The content in this release is the sole
responsibility of the authors and does not necessarily represent
the official views or imply endorsement of the National Institutes
of Health.
About University of Michigan, Metabolism, Endocrinology
and Diabetes Division
The University of Michigan, Metabolism, Endocrinology and
Diabetes Division and Brehm Center for Diabetes collectively house
a major referral for the study of lipodystrophy syndromes. For more
information, contact Adam Neidert at (734) 615-0539.
About Novelion Therapeutics
Novelion Therapeutics is a biopharmaceutical company dedicated
to developing new standards of care for individuals living with
rare diseases. The company seeks to advance its portfolio of rare
disease therapies by investing in science and clinical development.
Novelion has a diversified commercial portfolio through its
indirect subsidiary, Aegerion Pharmaceuticals, Inc.
CONTACT:
Amanda Murphy, Director, Investor Relations & Corporate
CommunicationsNovelion
Therapeutics857-242-5024amanda.murphy@novelion.com
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