US FDA accepts Genzyme's resubmission of Lumizyme
This article was originally published in Scrip
The US FDA has accepted Genzyme's resubmission of the BLA for its injectable Pompe disease treatment Lumizyme (alglucosidase alfa) seeking approval for the therapy produced at a 4000 litre scale, and has designated it as a class 2 response, which carries a six month review timeline. The user-fee date is on June 17th.
The agency issued Genzyme its second complete response letter for Lumizyme in November (scripnews.com, November 17th, 2009). The enzyme replacement therapy is a version of the company's current Pompe disease treatment, Myozyme (which received orphan designation) but is produced at a significantly larger scale, using a 4,000 litre bioreactor rather than a 160 litre facility.
An FDA advisory panel endorsed the new formulation in October 2008, but BLA approval has been delayed by an earlier complete response letter, which cited manufacturing problems at Genzyme's Allston plant and lack of agreement on a risk evaluation and mitigation strategy (REMS), postmarketing verification study and labelling.
Pompe disease is also known as glycogen storage disease type II and acid maltase disease. It is a neuromuscular, autosomal recessive metabolic disorder caused by a deficiency in alpha-glucosidase, which is needed to break down glycogen. The build up of glycogen causes progressive muscle weakness that can affect various body tissues, particularly the heart, skeletal muscles, liver and nervous system.
Myozyme is the only drug approved for the condition. However, Amicus Therapeutics and Shire have a drug candidate in, duvoglustat, Phase II development, which acts as an orally available pharmacological chaperone that stimulates alpha glucosidase.