Shire facility could answer rare disease drug shortage
pharmafile | November 27, 2011 | News story | Manufacturing and Production |Â Â Fabry disease, Gaucher, Shire, VprivÂ
Shire has filed applications with the European and US regulators to produce its Gaucher disease drug Vpriv (velaglucerase alfa) at a new manufacturing site in Lexington, Massachusetts, setting it on track to start boosting supplies to patients in 2012.
The company said the facility will give it flexibility to meet global demand for its other rare disease treatment, Replagal (agalsidase alfa) for Fabry disease, by freeing up manufacturing capacity at another plant in Alewife.
The Lexington facility already has approval from the EMA for the purification of Replagal, according to Shire.
“Accelerating the completion of our new manufacturing facility in Lexington will enable us to provide substantial additional capacity for Vpriv less than two years after launch,” commented Bill Ciambrone, senior vice president, technical operations at Shire’s Human Genetic Therapies division.
Additional capacity coming online is good news for Gaucher and Fabry patients, who have been hit by treatment shortages caused by manufacturing problems at Sanofi-owned rival company Genzyme.
Genzyme was forced to shut down a plant in Allston Landing temporarily in 2009 after a contamination incident, leading to enforced rationing and dosage restrictions for many patients on the company’s Gaucher drug Cerezyme (imiglucerase) and Fabrazyme (agalsidase beta) for Fabry disease.
While the company has been trying to rectify the situation by getting Allston Landing back up to code and transferring production to other sites in its manufacturing network, the process has proved challenging. In September, the company was forced to rein back its estimates for resuming supplies for Cerezyme because of a decrease in yields of the drug and longer release times for batches.
A report by analysts at Wells Fargo concluded earlier this month that Shire has not been able to fill the gap caused by Genzyme’s troubles, and this situation is unlikely to change until Shire’s Lexington facility comes online.
The consequences of shortages for patients are hard to gauge, but a recent study carried out in Dutch Fabry disease patients and published in the Orphanet Journal of Rare Diseases indicated that while no increase in clinical events had been seen as a result of the shortages, increases in biomarkers did suggest that patients were experiencing an increase in underlying disease activity.
A new Gaucher treatment from Pfizer and Protalix Biotherapeutics’ Uplyso (taliglucerase alfa) is currently under review by the FDA, and could provide further options for patients if approved.
Phil Taylor
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