VYNDAQEL

Key points

Favourable opinion for reimbursement in adult patients with wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM).

What therapeutic improvement?

Therapeutic improvement in the management of wild-type or hereditary transthyretin amyloid cardiomyopathy.

Role in the care pathway?

The care pathway for transthyretin amyloid cardiomyopathy is currently based on conventional symptomatic treatments for heart failure.

In the treatment of cardiac amyloidosis, three mechanisms of action are targeted:

• the elimination of abnormal transthyretin and/or its main synthesis source: a heart and/or liver transplant may be envisaged.
• stabilisation of transthyretin: tafamidis in the form of VYNDAQEL 20 mg, has a temporary recommendation for use (RTU) in hereditary or senile transthyretin amyloid cardiomyopathy and now has a marketing authorisation in France in the form of VYNDAQEL 61 mg.
• the resorption of toxic amyloid fibrils of transthyretin. 

In addition, another two medicinal products have a marketing authorisation in ATTR, but only in the neurological form: ONPATTRO (patisiran) and TEGSEDI (inotersen).

Role of VYNDAQEL in the care pathway:

VYNDAQEL (tafamidis) is a first-line treatment for transthyretin amyloid cardiomyopathy. It is the only medicinal product with a MA in this indication. The Committee highlights that:

• class NYHA IV patients had been excluded from the study and very few class NYHA I patients (8.4%) were included in the study,
• it questions the dose selected by the MA - 80 mg and not 20 mg - in the absence of robust data reported to the Transparency Committee making it possible to differentiate between the two dosages in terms of efficacy and safety.

For ATTR patients with both cardiac and neurological involvement, the Committee cannot currently reach a decision concerning the strategy of joint or sequential use of the various existing treatments - ONPATTRO (patisiran), TEGSEDI (inotersen) and VYNDAQEL 20 mg or now VYNDAQEL 61 mg (tafamidis) - in the care pathway for transthyretin amyloidosis with associated cardiomyopathy, in the absence of data.

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