Symkevi

tezacaftor / ivacaftor

Opinion

On 26 July 2018, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Symkevi, intended for the combination treatment of patients with cystic fibrosis (CF) aged 12 years and older. Symkevi was designated as an orphan medicinal product on 27 February 2017. The applicant for this medicinal product is Vertex Pharmaceuticals (Europe) Ltd.

Symkevi will be available as film-coated tablets containing 100 mg tezacaftor and 150 mg ivacaftor (ATC code: R07AX31). Symkevi is to be used in patients with F508del mutations affecting the CF transmembrane conductance regulator (CFTR) gene, which encodes for the CFTR protein. Tezacaftor facilitates the cellular processing and trafficking of the CFTR protein to the cell membrane, while ivacaftor improves CFTR protein function by increasing CFTR channel gating.

The benefits with Symkevi are its ability to improve pulmonary function and decrease the number and rate of pulmonary exacerbations in CF patients, measured over 24 weeks and 8 weeks in two pivotal studies. The most commonly observed adverse reactions are headache and nasopharyngitis.

The full indication is:

"Symkevi is indicated in a combination regimen with ivacaftor 150 mg tablets for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (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".

It is proposed that Symkevi be prescribed by physicians with experience in the treatment of CF. If the patient's genotype is unknown, an accurate and validated genotyping method should be performed to confirm the presence of an indicated mutation using a genotyping assay. As part of the combination regimen, Symkevi is to be taken in the morning and ivacaftor alone in the evening.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.

Key facts

Name
Symkevi
International non-proprietary name (INN) or common name
  • tezacaftor / ivacaftor
Active substance
  • tezacaftor
  • ivacaftor
Therapeutic area
Cystic Fibrosis
Additional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Orphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Date opinion adopted
26/07/2018
Company name
Vertex Pharmaceuticals (Europe) Ltd.
Status
Positive
Application type
Initial authorisation

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