Lowest Possible Ceredase/Cerezyme Dose Sought

The enormous cost of Genzyme's two Gaucher's disease therapies, Ceredase (alglucerase) and its recombinant form Cerezyme, requires continued effort to establish the lowest effective initial and maintenance doses, according to a US National Institutes of Health advisory panel on March 1.

The panel noted that the NIH should take the initiative and set up a cooperative team of researchers to address the issue, its three proposed tasks being: the establishment of a patient registry; analysis of the existing clinical data on response to treatment and natural history of the disease; and the design and conduct of clinical trials to address unanswered questions.

However, the proposals have attracted criticism from some clinicians who insist that they do not go far enough. For example, Ernest Beutler of the Scripps Research Institute believes there are simply not enough patients to make the proposed trials feasible and maintains that they would take too long - there is a desperate need for dosing recommendations now, he said.