Rare Disease Report

Price of Myalept to be $500K - $600K per Patient per Year

JANUARY 13, 2015
James Radke

Aegerion Pharmaceuticals – the makers of lomitapide (Juxtapid) to treat homozygous familial hypercholesterolemia (HoFH) - has completed its acquisition of metreleptin (Myalept), a drug approved in February 2014 by the U.S. Food and Drug Adminstration (FDA) as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. It acquired the drug from Astra Zeneca. Aegerion  paid $325 million upfront to acquire the global rights to develop, manufacture and commercialize metreleptin. In addition to the US, the drug is also approved in Europe and Japan.

During a JP Morgan healthcare conference presentation, Aegerion's chief executive officer Marc Beer noted that the drug was launched in June 2014 by Astra Zeneca with 8 sales reps with 35 patients currently on therapy (prevalence is approximately 1 in 1 million).

Since Aegerion has a sales force in place for its HoFH product to reach cardiologists and endocrinologists, the company is very hopeful their current representatives  can be used to market both lomitapide and metreleptin.  The company also plans to reach out to pediatric endocrinologists about metreleptin.

$500K - $600K Pricetag

During the JP Morgan presentation, Dr. Beer noted that Aegerion’s launch price for metreleptin will be $500-$600K per patient per year which he argued is a fair price based on value and prevalence of the condition.

Lipodystrophy is a group of rare syndromes characterized by loss of fat tissue. In some patients, it is genetic, and in others it may be acquired for different pathophysiological, and in some cases unknown, reasons. Generalized lipodystrophy is characterized by a widespread loss of fat tissue under the skin. This loss of fat tissue causes a deficit in the hormone leptin leading to multiple metabolic complications.

Metreleptin is only available through restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myalept REMS Program. Patients taking metreleptin may be at risk for developing anti-metreleptin antibodies with neutralizing activity or T-cell lymphoma.

Image courtesy wikimedia commons.

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