Genzyme says Cerezyme shortages could last longer than expected
pharmafile | August 17, 2009 | News story | Research and Development, Sales and Marketing |Â Â Gaucher, GenzymeÂ
The shortage of Genzyme's enzyme replacement therapy Cerezyme look set to last longer than expected, as the company has taken the decision to discard most of the in-process material produced at its Allston facility ahead of its contamination problems.
In Europe this will mean restricting availability of the drug to patients in the greatest need of treatment – guidance that is expected to remain in force to the end of the year.
Meanwhile, the US Food and Drug Administration (FDA) has asked Genzyme to provide details of the steps it is taking to restore supply, and will visit the Allston manufacturing plant again in the coming days to monitor progress. The biotechnology company is now predicting that shortages will extend through the end of the year.
Genzyme has decided to trash 80% of the material generated before the bioreactor contamination was uncovered in order to "minimise risk to the Allston plant and ensure that new supply will be available by the end of this year".
The company is still debating whether it should finish the remaining in-process material, and is discussing with the FDA whether it can ship two lots of Cerezyme that were produced before the contamination was discovered. The company now expects to start supplying Cerezyme from Allston in November and December.
Genzyme had been hoping to use some of the in-process material in order to restore supplies of Cerezyme (imiglucerase for injection) earlier and alleviate shortages of the drug, used to treat Gaucher disease, but now believes doing so could introduce a risk of perpetuating the viral contamination problems at the plant.
The decision means that the firm's second-quarter results have been re-worked to include an $8.4 million write-off charge in addition to the $14.2 million charge already announced.
"Reaching a decision on the work-in-process material has been difficult for us as we balance the medical benefit of Cerezyme for patients with minimizing the risk to our newly cleaned Allston plant," said Henri Termeer, chief executive of Genzyme, in a statement.
To prevent shortages in the future the company is constructing an additional manufacturing facility in Framingham, Massachusetts, which should be complete by the end of this year and validated and approved for production by mid-2011.
In the meantime, the FDA has given a green light to US doctors to prescribe a rival product from Shire – velaglucerase – which has not yet been approved for marketing.
Supplies of Cerezyme are being doled out only to those patients with the greatest clinical need.
Genzyme is also trying to expand access to its own experimental Gaucher disease drug – Genz-112638 – which is about the enter phase III testing.
There have been no changes to the anticipated supplies of Genzyme's Fabrazyme (agalsidase beta) for Fabry disease, another product affected by the contamination problem at Allston, the firm said.
Cerezyme – the European picture
Meanwhile in Europe Cerezyme is to be reserved for patients only in greatest need of treatment, following a severe shortage of the drug caused by manufacturing issues.
European regulator the EMEA has updated its recommendations for the product during the supply shortage, proposing the following measures:
* Infants, children and adolescents should receive Cerezyme at a reduced dose or at a reduced infusion frequency. However, no patient should be treated at a dose lower than 15 units per kilogram body weight every two weeks, or alternative treatment should be considered.
* Adult patients with severe, life-threatening disease progression should receive Cerezyme at a reduced dose or at a reduced infusion frequency. No patient should be treated at a dose lower than 15 U/kg every four weeks, or alternative treatment should be considered.
* In adult patients without severe, life-threatening disease, alternative treatment should be considered or treatment should be interrupted.
* All patients should be monitored for changes in haemoglobin, platelets and chitotriosidase levels, as appropriate, at baseline and bimonthly thereafter.
The EMEA updated its guidance after being told shortages of the product would be heavier than expected and the changes are expected to remain in place until the end of the year.
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