Genzyme has advised the EMEA that the supply shortages of Cerezyme, Fabrazyme are expected to continue at least until 09/10

Further supply disruption of Genzyme's Cerezyme and Fabrazyme leads EMEA to give new temporary treatment recommendations; but no problems with Myozyme

Article | 23 April 2010

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Ghislaine SURREL

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Troubled US biotech firm Genzyme has advised the European Union health regulator that the supply shortages of its rare disease drugs Cerezyme (imiglucerase) and  (agalsidase beta) are expected to continue at least until the end of September 2010, because of a new manufacturing problem.

Responding, the European Medicines Agency (EMEA) Committee for Medicinal Products for Human Use (CHMP) noted that temporary treatment recommendations to manage patients relying on these medicines during the ongoing supply shortages have been in place since June 2009 and have been regularly updated.

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Patients could be switched to Shire’s Replagal

For Cerezyme, the temporary treatment recommendations given on October 22, 2009 are still valid. However, for Fabrazyme, the CHMP decided to revise the recommendations made on 25 September 25, 2009, since, based on information supplied by the manufacturer, at least 12% of patients on the reduced Fabrazyme dose regimen have already experienced a worsening of their disease. For such patients, the CHMP recommended that physicians should either consider restarting the original treatment with the full dose of Fabrazyme or switching to an alternative treatment, such as Replagal (agalsidase alfa), marketed by UK drugmaker Shire.

Given the new information, the temporary treatment recommendations for Fabrazyme are as follows:

* Children and adolescents (<18 years) should receive fabrazyme according to the standard dose and frequency of one infusion every two weeks. >

* Adult patients already treated/stabilised may receive Fabrazyme with an adjusted dose of 0.3 mg/kg as maintenance dose every two weeks.

* All patients, especially those with adjusted dose regimens, should be under close clinical surveillance. A full medical examination, including all relevant clinical parameters, should be performed every two months. It is of the utmost importance to monitor the plasma GL-3 or urinary GL-3 levels, as for the moment the GL-3 level is the most sensitive parameter.

* For patients on the reduced dose who demonstrate a deterioration of the disease, physicians should consider restarting the original treatment with the full dose of Fabrazyme or switching to an alternative treatment, such as Replagal.

The new manufacturing problem has occurred at the production site in Allston Landing, in the USA, where Cerezyme and Fabrazyme are produced. It relates to a problem with the plant's water system. The defect has now been corrected, but supplies for both medicines will not return to normal before the end of September 2010, according to Genzyme. The company recently reported first-quarter financial results, which reflected the supply problems of the drugs in reduced turnover (The Pharma Letter April 22).

Because of a series of manufacturing difficulties since June 2009, the CHMP remains concerned about the continued supply shortages of Genzyme's medicines, and is, together with the European network, closely monitoring the situation. Genzyme has been invited to present its manufacturing quality assurance system to the Committee during the May plenary meeting. The Agency will make further updates as appropriate.

No safety concern with Myozyme

Additionally the Committee said it was informed by Genzyme Europe BV of a production problem at one of its manufacturing sites, the Flanders facility in Geel, Belgium, of Myozyme (alglucosidase alfa). During routine maintenance Genzyme discovered a faulty pump seal that could have potentially contaminated Myozyme batches with hydraulic oil.

After reviewing all available data, including results of the analysis of samples of the concerned batches using enhanced analytical methods that did not reveal any contamination, the Committee concluded that there was no concern about the safety of Myozyme, used in the treatment of Pompe disease. The problem has now been corrected and production is resuming. While currently no shortage of Myozyme is expected, the CHMP says it will be monitoring the availability of the drug on the market.

http://www.thepharmaletter.com/file/94416/further-supply-disruption-of-genzymes-cerezyme-and-fabrazyme-leads-emea-to-give-new-temporary-treatment-recommendations-but-no-problems-with-myozyme.html

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