FDA fast tracks Shire on Genzyme woes

Shire plc is reporting that the FDA will allow the company to distribute its treatment for Gaucher disease before FDA approval, to blunt the effect of a shortage of the only drug on the market, Genzyme’s Cerezyme.

UK based Shire, whose Human Genetic Therapies division is headquartered in Cambridge and Lexington, also released positive phase 3 data for the Gaucher drug, called velaglucerase alfa. The company also announced that it has begun filing portions of the New Drug Application for the medication, and will finish its submission by the end of this quarter.

“We are very pleased with the progress of the velaglucerase alfa program from both a clinical and regulatory perspective,” said Sylvie Gregoire, President of Shire Human Genetic Therapies. “This data are consistent with those previously reported from the Phase I/II and extension studies. We will continue to work diligently with the FDA and other regulatory agencies to make velaglucerase alfa available as soon as possible to help meet the needs of the Gaucher community.”

Shire officials say they are conducting the largest and most comprehensive set of Phase III clinical trials conducted to date for Gaucher disease. More than 100 patients at 24 sites in 10 countries around the world have participated in the clinical studies.

The first trial in the Phase III program to be completed was a multicenter, randomized, double-blind, two dose study of velaglucerase alfa in patients with Type 1 Gaucher disease. The primary goal of this study was to evaluate the safety and efficacy of velaglucerase alfa in 25 patients with Type 1 Gaucher disease.

The the first phase 3 trial, velaglucerase alfa was found to be generally well tolerated with no drug-related serious adverse events reported in the trial. No patients withdrew from the trial due to an adverse event. Most of the drug-related adverse events were reported in association with velaglucerase alfa infusions, all of which were mild and resolved without long-lasting effects.

The only Gaucher disease treatment currently on the market is Cerezyme, made by Cambridge-based Genzyme Corporation. Genzyme’s production cannot currently meet projected demand following a temporary closure at the company’s manufacturing facility in Allston, due to contamination by a virus. Genzyme has resumed production of the medicine but it takes four months to develop from start to finish.

Gaucher disease results from a specific enzyme deficiency in the body, caused by a genetic mutation received from both parents. This panethnic disease involves many organ systems, such as liver, spleen, lungs, brain, metabolism and bone marrow.

Worldwide, the diagnosed population of Gaucher Disease patients is approximately 7,000.