Ivacaftor (7) – Kalydeco®
Cystic fibrosis (CF), heterozygous for F508del, ≥ 12 years, combination with tezacaftor
Characteristics
| Start date | 01.09.2019 – Marketing authorisation: 10.10.2018 |
|---|---|
| Resolution | 20.02.2020 |
| INN | Ivacaftor |
| Brand name | Kalydeco® |
| Pharm. company | Vertex Pharmaceuticals (Germany) GmbH |
| G-BA Procedure ID | D-477 |
| ATC code | R07AX02 Other respiratory system products (R07AX) |
| ICD-10 codes (AIS) | E84.0Cystic fibrosis with pulmonary manifestations, E84.1Cystic fibrosis with intestinal manifestations, E84.80, E84.87, E84.88, E84.9Cystic fibrosis, unspecified |
| Alpha-ID codes (AIS) | I129376Neonatal hepatobiliary disease in cystic fibrosis, I130516Cystic fibrosis with other multiple manifestations, I18531Cystic fibrosis, I2487Cystic fibrosis with pulmonary manifestation, I2488Cystic fibrosis with intestinal manifestation, I32495Cystic fibrosis with manifestations in the lungs and digestive tract |
| ORPHAcodes (AIS) | 586Neonatal hepatobiliary disease in cystic fibrosis, 586Cystic fibrosis with other multiple manifestations, 586Cystic fibrosis, 586Cystic fibrosis with pulmonary manifestation, 586Cystic fibrosis with intestinal manifestation, 586Cystic fibrosis with manifestations in the lungs and digestive tract |
| DDD | 1 U O |
| Therapeutic area | Metabolic diseases Cystic fibrosis (CF) Orphan (turnover limit) |
| Reason for procedure |
Reassessment: Orphan turnover exceeded
Original resolution: Ivacaftor (2) (19.02.2015) |
| Specialty | Bundling |
| Therapeutic indication of the resolution |
|---|
|
In a combination regimen with tezacaftor 100 mg/Ivacaftor 150mg tablets for the treatment of patients aged 12 years and older with cystic fibrosis (CF) who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Patients 12 years of age and older with cystic fibrosis who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G and 3849+10kbC→T. | Best-Supportive-Care |
Studies and Results
|
No. of studies
(best subpopulation) |
1 (VX14-661-108) |
|---|---|
|
Study design
(best subpopulation) |
H2H vs. ACT |
|
Meta analysis
(best subpopulation) |
no |
- Clinical trials
- To assess the additional benefit of IVA + TEZ/IVA in patients aged 12 years and over with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and who have a mutation on the second allele with residual CFTR function (RF mutation) on the second allele, the pharmaceutical manufacturer submitted the Phase III multicentre, randomised, double-blind, placebo-controlled crossover study VX14-661-108 (hereinafter ‘Study 108’) was submitted by the pharmaceutical company.
Patients aged 12 years and over with cystic fibrosis who are heterozygous for the F508del mutation and carry one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G and 3849+10kbC→T
- The additional benefit is not proven.
- Consequently, the pharmaceutical manufacturer did not submit any study for this patient population that would have been suitable for assessing the additional benefit of IVA + TEZ/IVA compared with the appropriate comparator therapy.
- Overall assessment
- Overall, for patients aged 12 years and over with cystic fibrosis who are heterozygous for the F508del mutation and carry one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G and 3849+10kbC→T, for whom additional benefit is not proven for treatment with IVA + TEZ/IVA.
Courtesy translation only, please refer to the German original.
Associated procedures
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