Genzyme Corporation (GENZ) finally received some good news in the form of US Food and Drug Administration (FDA) approval for its Pompe disease treatment, Lumizyme (alglucosidase alfa). The FDA granted approval to Lumizyme, which has been manufactured at Genzyme’s 4000 liter bioreactor scale at the manufacturing facility in Geel, Belgium.
The approval process for Lumizyme has not exactly been smooth sailing for the company. Genzyme had initially submitted an application seeking approval for Lumizyme manufactured at its 2000 liter bioreactor scale at its Allston Landing manufacturing facility.
However, the FDA issued a complete response letter in November 2009 stating that the agency will not grant approval unless the company addresses the manufacturing issues at the Allston plant. Genzyme subsequently entered into discussions with the agency and stated that it will instead seek approval for the manufacture of Lumizyme at its 4000 liter-scale manufacturing plant in Geel, Belgium.
Lumizyme is marketed outside the US as Myozyme. Myozyme is approved for the treatment of Pompe disease in about 45 countries worldwide. Myozyme, manufactured using a 160 liter scale process, is available in the US for the treatment of infants and children.
Lumizyme is the first treatment to be approved in the US especially for the treatment of patients with late-onset Pompe disease. Lumizyme will be available in the US under a Risk Evaluation and Mitigation Strategy called the Lumizyme ACE (alglucosidase alfa control and education) Program. The product label contains a black-box warning regarding anaphylactic reactions, allergic reactions and immune reactions.
We note that Genzyme has been providing Lumizyme free of cost under its Alglucosidase Alfa Temporary Access Program (ATAP) to severely affected adults with Pompe disease. About 200 patients are currently receiving treatment under this program. Now that Lumizyme has been approved, Genzyme will be transitioning these patients to the commercial product over the next few quarters.
The approval of Lumizyme is the first good news announced by the company in a long while. Genzyme has been plagued with manufacturing and supply issues over the past few months. The company has yet to resume normal supply of key products, Cerezyme and Fabrazyme, and is facing a consent decree as well.
We currently have an Underperform recommendation on Genzyme. Genzyme’s financial results in 2009 were severely impacted by the temporary shutdown of the Allston manufacturing facility due to contamination issues. We believe that the company may have to face additional challenges before it is able to go back to a normal production and supply schedule.
We expect investor focus to remain on the company’s emerging pipeline, updates regarding the supply schedule of Cerezyme and Fabrazyme, and new guidance.
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