6 November 2014

AstraZeneca today announced that it has entered into a definitive agreement with Aegerion Pharmaceuticals, Inc. to divest Myalept™ (metreleptin for injection), an orphan product that is indicated to treat complications of leptin deficiency in patients with generalised lipodystrophy.

Myalept is the first and only product approved in the US for the treatment of generalised lipodystrophy and it has orphan drug designation in the US, EU, and Japan. Myalept is a recombinant analogue of human leptin, indicated in the US as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalised lipodystrophy.

Under the terms of the agreement, Aegerion will pay AstraZeneca $325 million upfront to acquire the global rights to develop, manufacture and commercialise Myalept, subject to an existing distributor licence with Shionogi covering Japan, South Korea, and Taiwan. The transaction does not include the transfer of any AstraZeneca employees or facilities.

Luke Miels, Executive Vice President, Global Product and Portfolio Strategy, AstraZeneca, said: "Generalised lipodystrophy is a rare condition with significant unmet medical need that can impact every aspect of a patient's health. Myalept is the first therapy to provide a real option for treating complications of this disease and we are pleased that patients will benefit from its progress under Aegerion as a company with expertise in rare diseases. The divestment of Myalept reinforces our focus on core strategic priorities and will allow us to concentrate our resources on disease areas where we can make the biggest difference to patients."

Marc Beer, Chief Executive Officer at Aegerion, said: "The therapeutic profile of Myalept is ideally aligned with Aegerion's commitment to bring innovative therapies to patients with rare diseases. We plan to apply our team's first-hand experience in bringing a novel therapy for a rare dyslipidaemia to patients who have previously had inadequate therapeutic alternatives. We expect the Myalept business to be highly synergistic with our current operations."

The divestment transaction is subject to closing conditions, including the receipt of antitrust clearance from the US Federal Trade Commission. The companies expect the transaction to complete in January 2015.

– ENDS –


About generalised lipodystrophy

Generalised lipodystrophy consists of a rare set of syndromes that are inherited or acquired through an autoimmune response and that are characterised by loss of fat tissue, typically from under the skin. The underlying reason is a deficiency in leptin, leading to an inability to store triglycerides in normal fat depots. This often leads to severe insulin resistance and diabetes, severe hypertriglyceridaemia along with a concomitant increased risk of acute pancreatitis, and hepatic steatosis, which can lead to cirrhosis.

About Aegerion

Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the development and commercialisation of innovative therapies for patients with debilitating rare diseases. For more information about the company, please visit

About AstraZeneca

AstraZeneca is a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of cardiovascular, metabolic, respiratory, inflammation, autoimmune, oncology, infection and neuroscience diseases. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit:


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