Cerezyme shortage

[chantell.reagan]

We received an anonymous question: "What’s the story with the Cerezyme shortage? Have plans encountered any shortage issues? If so, how are plans dealing with these issues?"

[chantell.reagan]

Back in June 2009, Genzyme discovered a virus in one of its manufacturing facilities, which produces Cerezyme (imiglucerase) for Gaucher disease. The company decided to halt production to remove the virus, but shipped finished lots that were held in inventory, determined not to be affected by the virus. Originally Genzyme estimated that the shortfall would end in October, but in the latest press release they announced that 80% of their supply material had to be discarded to avoid the risk of re-contamination, and new material should normalize Cerezyme supplyby end of this year (November or December 2009).

Cerezyme is the current standard of care for Gaucher disease type 1 and the shortage has serious implications for the Gaucher community. In June, the National Gaucher Association issued voluntary guidance for reserving Cerezyme for the most vulnerable population (i.e. infants, children, adolescents, and adults with progressing disease) but since the shortage is now projected to end by the end of the yea,r Genzyme has just implemented a dose conservation program based on this guidance.

The only other drug approved for this condition is Zavesca (miglustat) (which is an oral therapy taken 3 times/day); however, it’s indicated for adults with mild to moderate disease for whom enzyme replacement is not an option.

Also, there are 3 products in phase 3, which are being investigated as alternative enzyme replacement therapies for Gaucher disease. The manufacturers of these investigational therapies have petitioned the FDA (some based on the FDA’s request) to expand patient access through a treatment IND; so far, the FDA has approved Shire’s request and their drug will be initially provided free of charge to patients who are enrolled in the protocol:

o velaglucerase, IV alternative to Cerezyme investigated by Shire (expected to be reviewed by the FDA in the 1st half of 2010)
o prGCD (glucocerebrosidase), IV alternative to Cerezyme investigated by Protalix (expected to be reviewed in the 1st half of 2010)
o GENZ-112638, oral alternative to Cerezyme investigated by Genzyme (currently recruiting patients for 2 Phase III studies)

Additionally, this manufacturing issue affects another Genzyme product, Fabrazyme (agalsidase) for Fabry disease. The FDA will be requiring Genzyme to a contingency plan for Cerezyme, as well as Myozyme (alglucosidase alfa), used to treat Pompe disease.

Additional sources:

Genzyme press release 6/16/09
Genzyme press release 6/25/09
Genzyme supply update: Cerezyme
Protalix press release 7/09
Shire press release 8/09
Zavesca (miglustat) product page
News article 8/09

[chantell.reagan]

As of 8/18/09, the FDA approved the treatment protocol for prGCD, enabling healthcare professionals to administer the drug before it is approved by the FDA. The drug will be available free of charge to those enrolled in the protocol.

Source: News article 8/09