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16 Jul 2009
Message from Sylvie Grégoire, President, Human Genetic Therapies regarding the MLD experimental drug

16 Jul 2009 - We appreciate and fully respect the seriousness of Mr. Massart’s situation and empathize with his family’s heartache.

As we have explained to Mr. Massart several times since Shire acquired this development program, we are unable to produce enough of this experimental enzyme replacement therapy to provide it to any new patients outside of clinical trials.

Shire is currently working very closely with the contract manufacturer, which is the only company to ever make this experimental therapy, to produce it for clinical trials. Moreover, Shire is evaluating all avenues to increase production capacity for this investigational therapy, including the opportunity to bring the manufacture of it in-house.

In the press coverage of this unfortunate situation, there are some facts that have been neglected which we believe are important:

1. This experimental drug is not approved by any government authority. It has never been launched or marketed in any country in the world.

2. The safety of the drug is being tested in a very small clinical trial currently involving fewer than ten MLD patients. Trials aimed at testing if the drug is effective to combat MLD have not yet started – these always follow the safety study. Until these trials are completed and the safety and efficacy of the drug is known, Shire’s MLD drug cannot be considered a life-saving drug for Sandra or any patient suffering from MLD. We are working as fast as we can to initiate this trial and of course hope that the trial, which will take at least two years to complete, will be successful and will provide treatment to the 2,000 MLD sufferers worldwide. The start of the trial is dependant on the availability of supplies produced by the contract manufacturer.

3. Clinical trials are set up by doctors who specialize in MLD, and are independent of Shire. The ongoing trial currently involves fewer than ten children who were between the ages of 24 and 60 months at the start of the study.

4. We have met and spoken with Mr. Massart and his daughter’s physician several times. We have always been clear that we are unable to supply Shire’s MLD drug, due to supply constraints, to Sandra and indeed to any patient who is not currently on the drug. The reason for this is that the manufacturing process for MLD is very complex and our contract manufacturer is only able to supply very small quantities of the experimental drug. We have never promised Mr. Massart the drug; nor have we ever asked Mr. Massart for any money for the drug.

5. Our challenge in developing this experimental drug rests in the fact that producing it is particularly complex and requires a great deal of time. Due to the complexity associated with manufacturing, we are currently unable to offer access to this experimental drug to any new patients outside of the clinical trials.

We appreciate that our inability to offer access to our experimental drug is distressing and frustrating for all MLD patients and their families, but let me assure you that we are working diligently and with priority to bring a much needed therapy to MLD patients as soon as possible. Considering the above mentioned remaining timeline for development of the product, which are being kept as short as possible by working with the regulatory authorities, one could hope to have commercially available product for all MLD patients in three to five years.

Please be assured that we are doing all we can.

Kind regards,

Sylvie Grégoire, President, Shire HGT

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