Shire's Gaucher's drug meets primary endpoint in Phase III trial
This article was originally published in Scrip
Shire's Gaucher's disease drug velaglucerase alfa met its primary endpoint in a Phase III trial, according to preliminary data. In light of shortages of Genzyme's Gaucher's disease treatment Cerezyme (imiglucerase), the US FDA has accepted a treatment protocol for velaglucerase alfa, adds Shire, which will enable physicians to treat Gaucher's disease patients before the drug's commercialisation.
For now, Shire says it will provide velaglucerase alfa free to patients who are enrolled in the protocol.
Patients with Gaucher's disease, an autosomal recessive disease, have a deficiency in the enzyme glucocerebrosidase, resulting in a lysosomal storage disorder. The glucocerebrosidase Cerezyme is approved for type 1 Gaucher's disease, but Cerezyme production has been temporarily halted due to a viral contamination at the drug manufacturing plant. Shire's velaglucerase alfa is a human expressed glucocerebrosidase that is currently in Phase III development.
preliminary results
In the recently completed trial, 25 patients with type 1 Gaucher's disease were randomised to receive either 45U/kg or 60U/kg of velaglucerase alfa every other week for 12 months.
While detailed results are not yet available, the company says that the trial met its primary endpoint, with patients on the higher dose achieving a statistically significant increase in mean haemoglobin concentration compared with baseline. Various secondary endpoints, including platelet and spleen sizes, were also met.
The treatment was reportedly well tolerated and there were no drug-related serious adverse events.
"This data are consistent with those previously reported from the Phase I/II and extension studies," said Sylvie Grégoire, president of Shire Human Genetic Therapies.
Two other ongoing Phase III trials are examining the drug in more than 75 additional patients, with results expected later this year and next year.
Shire has begun a rolling submission of an NDA for its fast-tracked drug, and expects to complete the application by the end of the third quarter.
The US FDA has also asked Protalix Biotherapeutics to submit a treatment protocol for its plant-cell-expressed glucocerebrosidase prGCD, which is currently in Phase III development for Gaucher's (scripnews.com, July 7th 2009).