Therapy Areas: Hereditary Disorders
Vertex Inks New Portfolio Reimbursement Agreement in Italy Including Kaftrio, Symkevi and Additional Indications of Orkambi and Kalydeco for Eligible Patients with Cystic Fibrosis
25 June 2021 - - UK-based biotechnology company Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) has reached a new portfolio agreement with the Italian Medicines Agency, AIFA, for the reimbursement of all of Vertex's approved medicines for the treatment of cystic fibrosis, including Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, the company said.

Italian patients ages 12 years and older with one F508del mutation and one minimal function mutation or two F508del mutations in the cystic fibrosis transmembrane conductance regulator gene will now have access to Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor.

Additionally, under the terms of the agreement, eligible patients ages two years and older with CF who have two copies of the F508del mutation in the CFTR gene will now have access to Orkambi (lumacaftor/ivacaftor).

Eligible patients ages 12 years and older who either have two copies of the F508del mutation, or one copy of the F508del mutation and another responsive residual function mutation in the CFTR gene, will have broad access to Symkevi (tezacaftor/ivacaftor) in combination with ivacaftor.

The agreement also expands access to Kalydeco (ivacaftor) for eligible patients ages one year and older.

In addition, the agreement covers any new approved indication extensions for Vertex's CF medicines submitted and approved for reimbursement during the term of the contract.

Kalydeco was first reimbursed in Italy in 2015, followed by ORKAMBI in 2017 for patients 12 years and older.

Vertex's CF medicines are reimbursed in over 25 countries around the world including Australia, France, Germany, the Republic of Ireland, the Netherlands, Spain, Sweden, the UK and the US
About Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a Combination With Ivacaftor
Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg was developed for the treatment of cystic fibrosis in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.

Ivacaftor/tezacaftor/elexacaftor is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface.

The latest approved EU licensed indication for ivacaftor/tezacaftor/elexacaftor was supported by positive results of three global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study (Study 445-102) in 403 people with one F508del mutation and one minimal function mutation, a four-week Phase 3 study (Study 445-103) in 107 people with two F508del mutations, and a Phase 3 study (Study 445-104) in 258 people heterozygous for the F508del-CFTR mutation and a CFTR gating mutation or a residual function mutation.

Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface.

Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface.

Patients with two copies of the F508del mutation are easily identified by a simple genetic test.
Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

Ivacaftor is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene.

Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
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