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Condition update published on

Takeda to discontinue their TAK-609 clinical trial


It is with great sadness to announce that last week, Takeda announced that after 8 years, they would not be continuing with their trial of intrathecal enzyme replacement therapy (TAK-609). We know that those currently on the trial have been contacted by their clinical teams but also wanted to let all those who were enrolled previously know the news before posting publicly.

Unfortunately, Takeda was not able to demonstrate the endpoints required by regulatory authorities such as the FDA and EMA and the data was determined insufficient. Despite this Takeda has committed to providing the continued drug to those still on the trial until such time, as an alternative approved treatment option is available.

We have been asked to reassure those still enrolled on the trial that there will be no immediate change to how they receive their treatment. However, if you have any concerns please do speak to your treating clinician.

More information:

The MPS Society has requested a meeting for both current and previously treated patient families with Takeda. If this is something that you would be interested in attending please email Sophie Thomas here. The meeting will allow you the opportunity to hear directly from representatives of Takeda and to ask questions.

Both the RDRP and MPS Support and Advocacy Teams are here to support families affected by this news. If you have any queries in relation to the trial and logistics, please email Benedicta Marshall-Andrew here. If you need the support of the advocacy team, please email Steve Cotterell here.

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