Genzyme warned patients of Gaucher-drug rationing

Boston Business Journal – by Julie M. Donnelly

Genzyme Corp. will only ship the Gaucher disease treatment Cerezyme to children and the sickest of adult patients, according to an Aug. 3 letter to patients filed with the Food and Drug Administration. The move follows a disruption to the supply of the drug, caused by the six-week long shutdown of the company’s Allston, Mass., manufacturing plant due to contamination by a virus.

The letter to patients preceded the Cambridge, Mass.-based biotechnology company’s Aug. 10 announcement that it would have to discard 80 percent of the unfinished supply of Cerezyme that was in progress. That production was halted June 16.

A spokeswoman at the National Gaucher Foundation said in an interview that as early as Aug. 4, some patients who did not fit within the three most vulnerable categories, did not receive their medicine. Those categories were children under the age of 18; patients with types 2 or 3 Gaucher disease; or those with life threatening clinical situations.

Patients with Type 1 Gaucher disease, the most common form, are not eligible to receive the drug. The foundation spokeswoman said it was difficult to determine how many Gaucher patients will have their treatment regimens disrupted. Most patients receiving Cerezyme receive doses every two to four weeks, depending on their disease progression.

The spokeswoman said it was also difficult to gauge the impact on the affected patients’ health, because the progression of the disease is different in each patient.

Prior to the letter, patients were being asked to voluntarily forgo doses of Cerezyme if they were able to do so. The National Gaucher Foundation said that while many patients had been willing to observe these guidelines, regulatory restrictions and difficulty transporting the medication, which must be refrigerated, created obstacles.

In its Aug. 3 letter, Genzyme (Nasdaq: GENZ) thanked patients for their conservation efforts but said the company was still “at risk of not maintaining enough product to enable the most vulnerable patients to continue receiving doses through the constraint period.”

Genzyme officials have said the shortage will last until the end of the year.

Gaucher disease results from a specific enzyme deficiency in the body, caused by a genetic mutation received from both parents. The 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.