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Cerezyme Supply Update

20 August 2009

Dear Friends, I am writing to you, at what is a very difficult time for the global Gaucher community, to provide an update on the supply of Cerezyme and how the matter is being dealt with in the UK.

As you will no doubt be aware, on the 13th/14th June, a virus was found at the Genzyme facility in the United States used for the production of Cerezyme. Although there is no evidence that the virus affects humans, the plant was shut down and production was halted whilst the plant was sanitised. Production has restarted and it is anticipated that the first supplies of the new product will commence in mid November with full supplies of Cerezyme being available from the beginning of next year.

It was initially anticipated that the impact of the shutting of the plant would be to limit supplies of therapy (globally) to 60% of those previously supplied. This assumed that the material that was in the course of production could be used for patients. However in order to use much of this material it would have been necessary to reintroduce it into the sanitised plant. Genzyme decided not to do this for fear that the material may be contaminated, which would have resulted in the facility having again to be closed and resanitised. The effect of this decision is that 80% of the Cerezyme in production could not be used after all.

On the 10th August Genzyme decided that if supplies were to be available to the most needy patients that the global supply would need to be cut to approximately 20% of the previous normal dose until the end of the year. This of course has very serious and immediate consequences for all patients.

Genzyme have produced a slide presentation setting out the sequence of events which explains the situation in detail, you can view this on our homepage.

The European Medicines Agency (EMEA) have issued a press release which offers guidelines on the allocation of the Cerezyme which is now available. They recommend that priority is given to children, neuronopathic patients and adult patients with severe life threatening progressive disease. A copy of this press release can be viewed on our homepage.

In the UK we are very fortunate that the management and treatment of Gaucher Disease (as well as other Lysosomal Storage Disorders) is designated nationally by the National Commissioning Group (NCG). Doctors from the centres together with Dr Edmund Jessop (medical advisor to the NCG) have been holding regular teleconferences to formulate policy on a National basis to determine how best and most equitably to allocate the limited supply of Cerezyme. Daniel Brown and I have also participated these meetings.

A national plan is being developed to ensure that the must vulnerable patients receive priority but this does mean that all patients receiving treatment will see a dose reduction and many patients will not receive any treatment at all for some months. This is clearly not desirable but is unavoidable.

We have also been working with your doctors to explore alternative therapies. As you will know Zavesca (Miglustat) is an oral therapy which is approved for use in mild to moderate Gaucher disease. There are possible side effects but the EMEA do make reference to Zavesca in their guidelines and I do know that some doctors are considering this for their patients.

You will also be aware of the ongoing (and fairly advanced) clinical trials for two new enzyme therapies (Velaglucerase from Shire and prGCD produced by Protalix). Neither therapies yet have a product licence but both have received approvals from the US Food and Drug Administration (FDA) for their protocols for use. As yet, we do not know whether either company has any availability of product, and together with the NCG and your doctors we are seeking further information on whether either of these trial drugs could be offered to patients. In addition Genzyme are running trials of their "small molecule" therapy (GENZ-112638) which may be considered further.

I do know that your Doctors, and their teams from the centres, have been contacting patients to discuss the situation and the implication on individual treatment regimes. This is a very difficult and time consuming process and on behalf of you all I want to thank them for the dedication and compassion they have shown and for the sheer hard work this has entailed.

We are also working very closely with patient groups throughout the world, and in particular the National Gaucher Foundation in the United States, as well as the members of the European Gaucher Alliance. We are all in this together and are trying to work together to establish common policies.

We will keep you informed as more information becomes available. If you have any questions do contact us (email is preferable during the holiday period). Obviously if you have any medical concerns or particular questions about your treatment these must be directed to your doctor.

Yours

Jeremy Manuel O.B.E.
Chairman Gauchers Association (UK)