Aegerion Pharmaceuticals, Inc. Pays $325 Million Upfront For AstraZeneca PLC's Myalept™

November 6, 2014

By Krystle Vermes, BioSpace.com Breaking News Staff

Cambridge-based Aegerion Pharmaceuticals has announced that it will acquire orphan drug Myalept from AstraZeneca for approximately $325 million. Myalept, which is also known as metreleptin, is an orphan product designed to treat complications of leptin deficiency in patients with generalized lipodystrophy. It is the only approved product in the U.S. for the treatment of this condition.

Under the terms of the agreement, Aegerion will also receive the rights to develop, manufacture and commercialize the drug around the world. The transaction does not include the transfer of any AstraZeneca employees or facilities.

"Generalized lipodystrophy is a rare condition with significant unmet medical need that can impact every aspect of a patient's health,” said Luke Miels, executive vice president of Global Product and Portfolio Strategy at Astra Zeneca. “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."

Myalept is a recombinant analogue of the human leptin. It is adjunct to diet as a replacement therapy to treat complications of leptin deficiency. The companies expect the transaction to close by the end of January 2015.

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

Generalized lipodystrophy is characterized as a loss of fat tissue, typically under the skin. Leptin deficiency leads to an inability to store triglycerides in normal fat deposits. As a result, patients often develop insulin resistance and hypertriglyceridemia, as well as a risk of acute pancreatitis and hepatic steatosis.

At the moment, Myalept is not recommended for patients with HIV-related lipodystrophy or those who have metabolic disease, such as diabetes mellitus and hypertriglyceridemia.