04.22.08
The FDA has informed Genzyme that Myozyme (alglucosidase alfa) produced at the 160L bioreactor scale and Myozyme produced at the 2000L scale should be classified as two different products because of differences in the carbohydrate structures of the molecules. Genzyme currently has U.S. approval to sell Myozyme manufactured at the 160L scale and has been seeking FDA clearance for the 2000L scale. Production at this larger scale has been approved in more than 40 countries.
Based on the global clinical experience of nearly 900 patients currently receiving Myozyme produced at the larger scale, Genzyme believes that Myozyme produced at both the 160L and 2000L scales is clinically effective and safe. Myozyme is the only treatment for Pompe disease is a rare, progressively debilitating and life-threatening inherited disorder.
The FDA will require Genzyme to submit a separate BLA to gain approval for the 2000L scale. The company expects the FDA to give the BLA priority review and to act on the application by the end of this year, in which case, commercial 2000L Myozyme could be available in the U.S. during 1Q2009.
In May 2007, Genzyme in collaboration with the FDA created the Myozyme Temporary Access Program (MTAP) for severely affected adults with Pompe disease in the U.S.
"We are extremely disappointed in the FDA's decision because it will further delay broad patient access to Myozyme, which is not possible under the MTAP program," said Henri A. Termeer, Genzyme's chairman and chief executive officer.
As a result of the delay in the approval of 2000L production, 2008 Myozyme sales are expected to be approximately $275-$285 million compared to previous guidance of $320-$330 million.
Based on the global clinical experience of nearly 900 patients currently receiving Myozyme produced at the larger scale, Genzyme believes that Myozyme produced at both the 160L and 2000L scales is clinically effective and safe. Myozyme is the only treatment for Pompe disease is a rare, progressively debilitating and life-threatening inherited disorder.
The FDA will require Genzyme to submit a separate BLA to gain approval for the 2000L scale. The company expects the FDA to give the BLA priority review and to act on the application by the end of this year, in which case, commercial 2000L Myozyme could be available in the U.S. during 1Q2009.
In May 2007, Genzyme in collaboration with the FDA created the Myozyme Temporary Access Program (MTAP) for severely affected adults with Pompe disease in the U.S.
"We are extremely disappointed in the FDA's decision because it will further delay broad patient access to Myozyme, which is not possible under the MTAP program," said Henri A. Termeer, Genzyme's chairman and chief executive officer.
As a result of the delay in the approval of 2000L production, 2008 Myozyme sales are expected to be approximately $275-$285 million compared to previous guidance of $320-$330 million.