AMDA Letter to Patients Regarding Supply Situation
By: Tiffany House--AMDA President posted on: January 17, 2009 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...
AMDA Statement Regarding the Management of “Myozyme” supply
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...
PRESS RELEASE Priority access for children during Myozyme supply shortage
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 alfa), from Genzyme, during the expected supply shortage of the medicine over the...
IPA Statement Regarding The Management Of Myozyme Supply – Adivce To Pompe Patients
By: IPA posted on: January 14, 2009 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;...
IPA Statement Regarding the Management of Myozyme Supply
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...
IPA Statement Regarding the Management of Myozyme Supply – Adivce To Pompe Patients
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...
Guidance to the Pompe Community on the Management of Myozyme Supply
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...
MYOZYME® (2000 L) Supply Update – US Version – January 2009
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...
Myozyme Supply Update January 2009
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...
Genzyme Pompe Program Update—November 2008
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 disease. Prior to approval, the company and the agency first...
Genzyme Pompe Program Update—November 10, 2008
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 Begins Phase 2 Clinical Trial of AT2220 in Pompe Disease
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 trial of AT2220 (1-deoxynojirimycin HCl),...
Genzyme Pompe Program Update—April 2008
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...
January 2008 US Myozyme Supply Update
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...
Genzyme Study of Myozyme® for Late-Onset Pompe Patients
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...
Genzyme Pompe Program Update—October 2007
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MTAP Fact Sheet June 2007
MTAP, the Myozyme® (alglucosidase alfa) Temporary Access Program, is a clinical program to provide access to Myozyme produced by a larger scale manufacturing process for a limited time until this manufacturing scale is approved by the FDA. Patients enrolled in MTAP...
Genzyme Pompe Program Update—June 2007
Genzyme would like to take this opportunity to provide a brief update to the Pompe patient community regarding the US supply of Myozyme® (alglucosidase alfa). As planned, Genzyme submitted the necessary documentation to the FDA this month for the licensure of the...
MTAP Fact Sheet May 2007
MTAP, the Myozyme® (alglucosidase alfa) Temporary Access Program, is a clinical program to provide access to Myozyme produced by a larger scale manufacturing process for a limited time until this manufacturing scale is approved by the FDA. Patients enrolled in MTAP...
Genzyme Pompe Program Update—April 2007
As many of you are aware, a standard part of the biologics drug development process is to incrementally scale-up manufacturing capacity as demand for the product increases. During the development of Myozyme®, Genzyme began the manufacturing process using a smaller...