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Opinions on drugs - Posted on Apr 11 2012

Reason for request

Inclusion on the list of medicines refundable by National Health Insurance and approved for use by hospitals.


Clinical Benefit


The actual benefit of VYNDAQEL is moderate.

Clinical Added Value


VYNDAQEL offers a minor improvement in actual benefit (IAB level IV) in the management of adult patients with transthyretin (TTR) amyloidosis, given the lack of a treatment alternative. The Committee considers that VYNDAQEL can, at an early stage of the disease, delay the peripheral neurological impairment in symptomatic polyneuropathy patients. This effect does, however, appear to be on a modest scale and remains to be confirmed.

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