RUCONEST (conestat alpha)

Opinions on drugs - Posted on Mar 31 2021

Reason for request

New indication

Key points

Favourable opinion for reimbursement in the treatment of acute angioedema attacks in children aged 2 to 14 years with hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency.

Favourable opinion for reimbursement in adolescents aged over 14 years and adults in this indication.

What therapeutic improvement?

No clinical added value in the therapeutic strategy.

Role in the care pathway?

The management of HAE attacks is based on the administration of either icatibant (FIRAZYR) as a subcutaneous injection, or human plasma-derived (BERINERT, CINRYZE) or recombinant (RUCONEST) C1 inhibitor concentrate as a slow intravenous infusion. The more rapidly these are administered after the onset of the first symptoms of the attack, the more effective they are.

Role of the medicinal product in the care pathway

Given the data available in children aged 2 years and above and precautions which are not modified in adults and adolescents, the Committee considers that the characteristics of conestat alfa (RUCONEST), extracted from milk of transgenic rabbits and potentially containing traces of rabbit proteins, require that patients should be queried about prior exposure to rabbit proteins and informed about the signs and symptoms suggestive of an allergic reaction. This should be taken into account in the context of emergency administration and means that it is not a first-line therapy, if an alternative is available, in adults, adol

Clinical Benefit

Substantial

The Committee deems that the clinical benefit of RUCONEST (conestat alfa) in the MA indication:

is substantial in children aged 2 years and above,
remains substantial in adults and adolescents.

Clinical Added Value

no clinical added value

Considering:

the clinical data available, i.e., a phase 2, non-comparative, open-label study having included 20 children aged 5 to 14 years, which does not make it possible to specifically quantify the efficacy of RUCONEST (conestat alfa) in children aged 2 years and above compared to the available alternatives,
the characteristics of conestat alfa, which may contain traces of rabbit protein, thus requiring precautions to be taken prior to its administration in an emergency context,
the medical need met by the available alternatives, although these are subject to a risk of stock shortages,

the Committee considers that RUCONEST (conestat alfa) provides no clinical added value (CAV V) compared to the other treatments for acute HAO episodes available in children and adolescents aged 2 to 14 years.