AstraZeneca Receives FDA Approval for Orphan Drug

By Pharma News - DCAT Publisher

February 25, 2014

AstraZeneca has received FDA approval for its new chemical entity, the orphan drug Myaleptâ„¢ (metreleptin for injection), which is indicated as an adjunct to diet as replacement therapy for the treatment of complications of leptin deficiency in patients with congenital or acquired generalised lipodystrophy. Myalept, a recombinant analogue (laboratory-created form) of human leptin, is the first and only treatment approved by the FDA for these patients, according to AstraZeneca.

Lipodystrophy is a group of rare syndromes characterized by loss of fat tissue. In some patients it is genetic, while in others it may be acquired for different pathophysiological, and in some cases unknown, reasons. Generalized lipodystrophy is characterized by 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.

AstraZeneca is working to complete the transfer of the biologics license application (BLA) for Myalept from Bristol-Myers Squibb Company to AstraZeneca as part of the acquisition of the diabetes alliance assets, including Myalept and Amylin Pharmaceuticals, which was completed on February 1, 2014.

Source: AstraZeneca

 

 

 

 

 

 

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM386685.pdf

 

 

 

 

 

 

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM386685.pdf

 

 

 

 

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM386685.pdf

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