KALYDECO

Cystic fibrosis - First assessment (25 mg) and new indication (50 and 75 mg)

Opinions on drugs - Posted on Jun 29 2020

Reason for request

Inclusion and New indication

First assessment (25 mg).

New indication (50 and 75 mg).

Favourable opinion for reimbursement in the treatment of infants aged at least 6 months, toddlers and children weighing 5 kg to less than 25 kg with cystic fibrosis (CF) who have one of the gating (class III) mutations in the CFTR gene mentioned in the MA.

What therapeutic improvement?

Therapeutic improvement in the treatment of cystic fibrosis in infants aged at least 6 months to 1 year and weighing 5 kg to less than 25 kg who have one of the gating (class III) mutations in the CFTR gene mentioned in the MA.

Role in the care pathway?

The management of cystic fibrosis patients requires the intervention of a multidisciplinary team. Symptomatic treatment, which needs to be life-long, is based on 4 complementary symptomatic interventions:

respiratory management: physiotherapy, inhaled dornase alfa (which cannot be administered in patients under the age of 5 years), antibiotic therapy,
nutritional and gastrointestinal management,
implementation of optimal prevention of lung infections in accordance with the immunisation schedule,
patient education.

Role of the medicinal product in the care pathway

As in children aged 12 months and over, KALYDECO (ivacaftor) is a long-term treatment for cystic fibrosis, which should be prescribed from the outset in cystic fibrosis patients aged at least 6 months to 12 months and weighing 5 kg to less than 25 kg, with one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

The optimal treatment duration for KALYDECO (ivacaftor) is not known.

Special recommendations

Given the product characteristics and the complexity of treatment, the Committee reiterates that KALYDECO (ivacaftor) may only be prescribed by physicians with experience in the treatment of cystic fibrosis and recommends that patients be managed in reference centres specialising in the disease.

Clinical Benefit

Substantial

The clinical benefit of KALYDECO (ivacaftor) is substantial in the MA indication for the 25 mg strength and in the MA indication extension for the 50 mg and 75 mg strengths.

Clinical Added Value

important

Considering:

an efficacy and safety of ivacaftor (KALYDECO) in children aged at least 6 months to 1 year similar to those already assessed by the Committee in children aged from 1 year and over to less than 2 years, based on:
the results of a phase III non-comparative trial having essentially assessed the pharmacokinetics (primary endpoint) and safety of ivacaftor in children aged at least 6 months to 1 year,
the results of secondary or tertiary biological, symptomatic, growth parameter and palatability endpoints derived from exploratory descriptive analyses in this study,
the safety profile of ivacaftor, which appears to be acceptable in children aged from at least 6 months to 1 year,
the identified medical need in the absence of any other treatment targeting the causes of the disease,

and despite:

the follow-up limited to 24 weeks of treatment not enabling evaluation of the long-term efficacy and safety,

the Transparency Committee considers that, as in children aged over 1 year, KALYDECO (ivacaftor) provides an important clinical added value (CAV II) in the treatment of cystic fibrosis in infants aged at least 6 months to 1 year and weighing 5 kg to less than 25 kg who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.