Novelion Therapeutics Announces Marketing Authorization for MYALEPTA® (metreleptin) in the European Union to Treat Generalized and Partial Lipodystrophy
MYALEPTA, a recombinant analog of human leptin, is the first and only licensed treatment in
Pricing and reimbursement negotiations with healthcare authorities have commenced and will be pursued on a country-by-country basis.
"The approval of MYALEPTA in the EU is a significant advancement for patients with generalized and partial lipodystrophy and a significant milestone for our company,” said
The clinical data supporting the authorization resulted from an open-label, single-arm study evaluating MYALEPT treatment in 48 patients. In accordance with the receipt of a marketing authorization under exceptional circumstances, certain risk minimization measures and post-authorization obligations will be required, including proposed studies which will further the understanding of MYALEPTA’s impact on patients with generalized and partial lipodystrophy.
LD syndromes are ultra-rare disorders characterized by the irreversible loss of adipose tissue. Lipodystrophy can be genetic or acquired after an immune condition and occurs in childhood or adolescence. 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. Generalized lipodystrophy affects approximately one person out of every one million, whereas partial lipodystrophy affects approximately three people in every one million.
MYALEPT, the U.S. brand name of metreleptin, received approval from the U.
IMPORTANT SAFETY INFORMATION
Highlights of Safety Information from U.S. Prescribing Information for MYALEPT (metreleptin U.S. brand name)
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.
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 MYALEPT 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.
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 for MYALEPT.
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 securities laws. Any statements contained herein which do not describe historical facts, including expectations for pricing and reimbursement approvals in the EU, access to MYALEPTA in the EU, the size of the market for MYALEPTA in the EU, the potential market size for MYALEPTA, converting early access patients to commercial patients and adding new MYALEPTA patients in the EU, are forward-looking statements which involve risks and uncertainties that could cause actual results to differ materially from those discussed in such forward-looking statements. Such risks and uncertainties include, among others, those risks identified in our filings with the
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Source: Novelion Therapeutics, Inc.