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IPA Statement Regarding the Management of Myozyme Supply – Advice to Pompe Patients

Author: 
IPA
Category: Archive
Published
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; whether through their commercial or compassionate-use programs. Regretfully, in July 2008 Genzyme released a notification that supply would be tight in 2009 until a larger-scale production facility in Belgium is approved by the European Medicines Agency (EMEA).

Ever since that time, the IPA has urged Genzyme to find ways of increasing Myozyme production and, with the prospect of a tight supply, has worked very closely with Genzyme and expert physicians to prepare advisory guidelines for treating physicians. Playing an important role in the Myozyme Stakeholder’s Working Group (MSWG), the IPA has helped to formulate guidance for physicians, carefully designed to protect the most vulnerable patients. At this time, full protection can only be achieved by a reduction in demand for Myozyme from those patients who can tolerate a temporary interruption in their treatment.

Please click here to read more (PDF, 1.04 MB).

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