Genzyme Pompe Program Update- November 17, 2008 We would like to take this opportunity to provide an important update to the Pompe Community. The US Food and Drug Administration (FDA) has informed Genzyme that the FDA plans an accelerated approval for alglucosidase...

Myozyme Produced at the 2000 L Bioreactor Scale to Receive Accelerated Approval

Genzyme Program Update November 10th, 2008 Genzyme would like to take this opportunity to provide an update to the Pompe Community. The US Food and Drug Administration (FDA) held a meeting of its Endocrinologic and Metabolic Drugs Advisory Committee on October 21,...

New video added to “Meet the Patients”

A compilation of the most pertinent information regarding the Myozyme supply issue. The IPA has done its best to gather the most relevant and commonly asked questions regarding this matter. Please download the pdf file. Download complete article

The AMDA is writing to our Pompe patient community to let everyone know that the AMDA has been working closely with the IPA and Genzyme in order to get communication out to our patient population about the status of the Myozyme supply issue. Download complete...

The European Medicines Agency’s (EMEA) Committee for Medicinal Products (CHMP) has recommended that infants, children and adolescents be given priority access to Myozyme (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme.), from Genzyme, during the expected supply shortage of the medicine over the...

Since the first market approval of Myozyme, the IPA has liaised directly with Genzyme LSD Therapeutics to insist that an adequate inventory of the product was kept to ensure continuous supply to all patients receiving the treatment; whether through their commercial or...

Since the first market approval of Myozyme, the IPA has liaised directly with Genzyme LSD Therapeutics to insist that an adequate inventory of the product was kept to ensure continuous supply to all patients receiving the treatment; whether through their commercial or...

This guidance is being distributed to all treating physicians worldwide except those in the European Union (EU). Separate guidance will be sent to European healthcare professionals in accordance with the requirements of the European Medicines Agency (EMEA); we expect...

We would like to take this opportunity to provide an important update to the Pompe Community. Background In the early stages of development of Myozyme® (alglucosidase alfa), Genzyme initiated manufacturing at the 160 liter (L) scale, and has since scaled-up the...

We would like to take this opportunity to provide an important update to the global Pompe Community. Background In the early stages of development of Myozyme® (alglucosidase alfa), Genzyme initiated manufacturing at a small scale, and has since scaled-up the process...

The US Food and Drug Administration (FDA) has informed Genzyme that the FDA plans an accelerated approval for alglucosidase alfa produced at the 2000 L bioreactor scale for the treatment of late onset Pompe diseaseA form of Pompe disease that begins after infancy and usually progresses more slowly.. Prior to approval, the company and the agency first...

The US Food and Drug Administration (FDA) held a meeting of its Endocrinologic and Metabolic Drugs Advisory Committee on October 21, 2008 to discuss Genzyme’s Biologics License Application (BLA) for alglucosidase alfa produced at the 2000 L bioreactor scale to treat...

Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, today announced that it has initiated a Phase 2 clinical trialA research study that tests new treatments or approaches in people. of AT2220 (1-deoxynojirimycin HCl),...

We would like to take this opportunity to provide an important update related to Myozyme (alglucosidase alfa) manufacturing in the United States.  The FDA has informed Genzyme of its decision on the application for the larger scale manufacturing process to...

In April 2007, Genzyme implemented measures to manage the limited supply of Myozyme® (alglucosidase alfa) in the United States in order to ensure uninterrupted treatment for as many patients as possible.  Since April, both the patient and physician communities have...

CAMBRIDGE, Mass. – Genzyme Corp. (Nasdaq: GENZ) announced today that its Late Onset Treatment Study (LOTS) of Myozyme® (alglucosidase alfa) met its co-primary efficacy endpoints.  The study was undertaken to evaluate the safety and efficacy of Myozyme in juvenile and...