ADM 007-09-12/10
Genzyme Ataluren in nmDBMD Programme Update
December 2010
Genzyme have developed this Programme Update as a way to communicate about
ataluren and as part of our ongoing commitment in keeping the DBMD community
informed. For more information please feel free to contact Genzyme’s medical
information department at eumedinfo@genzyme.com. Please keep in mind that
certain national regulations in Europe may prevent any form of communication
between industry and patients (including the provision of non-promotional product
information) so in some cases a physician may need to inquire on a patient’s behalf.
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Genzyme and PTC continue to work diligently with regulatory authorities in different
regions regarding ataluren. Due to regional variances, the timing of these
interactions, procedures and pathways differ depending on the regulatory framework
for interaction between industry and authorities.
As you are aware, these differences have been highlighted by recent developments
in the USA, whereby PTC, with the support of the US FDA, implemented an open
label safety study as an alternative to supporting individual patient IND requests (a
US specific mechanism to gain access to non-approved treatments). Only nmDBMD
patients who previously participated in ataluren trials at U.S. sites are eligible for this
U.S. program. This particular safety study is not available in Europe.
We recognize that European families may be frustrated by a perceived lack of
progress in Europe in comparison to the US, and the difficulties in setting
expectations around the potential and timing of nmDBMD patients having access to
ataluren. Genzyme wants to assure you that we have been active in evaluating how
best to advance the programme and that we are making progress. We have recently
initiated meetings with EU regulatory authorities to seek scientific advice on the data
and potential paths forward, including the possibility of pre-approval access
programmes. Upon receipt of their formal feedback we will be in a position to
complete our evaluation and communicate next steps.
We appreciate your continued patience and understanding during this period of time
as we diligently work to complete our evaluation to determine the path forward. Our
commitment remains to update the nmDBMD community in the first quarter of 2011
and our goal is to make decisions that are in the best interest for this community.
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