Print“AstraZeneca acquired Myalept as part of its December 2013 deal to buy Bristol-Myers Squibb’s interests in the companies’ diabetes alliance for $2.7 billion.”
The U.S. Food and Drug Administration has approved AstraZeneca's Myalept, the first therapy for treating complications associated with a hormonal deficiency that can lead to insulin resistance and pancreatic damage.
The hormone, leptin, is made by fat tissue and patients with very little fat tissue have very low leptin levels. Leptin regulates food intake and other hormones, such as insulin. Patients with an unusual fat tissue shortage, a condition called lipodystrophy, often develop severe insulin resistance at a young age and may have diabetes mellitus that is difficult to control or very high levels of triglycerides in the blood that can lead to inflammation of the pancreas.
AstraZeneca acquired Myalept as part of its December 2013 deal to buy Bristol-Myers Squibb’s interests in the companies’ diabetes alliance for $2.7 billion. The deal included assets and personnel from Myalept developer Amylin Pharmaceuticals as well as $1.4 billion in potential regulatory, launch, and sales-related milestone payments. What part of the milestone payments may have been tied to the Myalept approval was undisclosed.
Myalept is an analog of leptin. The therapeutic was evaluated in an open-label, single-arm study that included 48 patients with congenital or acquired generalized lipodystrophy who also had diabetes mellitus, very high levels of triglycerides in the blood, and/or elevated levels of fasting insulin. The trial showed reductions in HbA1c—a measure of blood sugar control, fasting glucose, and triglycerides.
Because of the risk that certain patients taking the therapy could develop neutralizing antibodies and lymphoma, it will be available only through a FDA-sanctioned Risk Evaluation and Mitigation Strategy program. The program mandates certification and training of prescribers. The FDA is also requiring seven post-marketing studies for Myalept, including a long-term prospective observational study of patients treated with the therapy to assess immune responses and an assessment and analysis of spontaneous reports of potential serious risks related to the use of the drug. Eight additional studies are being requested as post-marketing commitments.
Mary Parks, deputy director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research says the drug “provides a needed treatment option for patients with this orphan disease.”
February 28, 2014
http://www.burrillreport.com/article-fda_approves_astrazeneca_therapy_for_rare_disease.html
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