Vertex CF Treatments Opening to Eligible Patients in France and Italy 

Vertex Pharmaceuticals has reached reimbursement agreements with health authorities in France and Italy for its various cystic fibrosis (CF) treatments, including Kaftrio (ivacaftor/tezacaftor/elexacaftor), a next-generation therapy approved for use with ivacaftor.

With these agreements — which bring the treatments into each country’s public health program — about 3,000 CF patients in France and Italy will gain access for the first time to more or all of Vertex’s CFTR modulators. Kaftrio — marketed as Trikafta in the U.S. — is estimated to treat 90% of all patients.

CF is caused by two defective copies of the CFTR gene. Such mutations lead to a dysfunctional CFTR protein, resulting in a poor flow of salt ions and water in and out of cells in organs, particularly the lungs. This gives rise to the buildup of abnormally sticky mucus that can cause progressive lung damage and trigger chronic lung infections.

Most CF patients carry at least one F508del mutation, which results in non-working or not enough CFTR proteins at the cell surface.

The reimbursement agreement with French authorities gives broad access to Kaftrio in combination with 150 mg of ivacaftor — sold as Kalydeco — to CF patients ages 12 years and older with one F508del mutation and another mutation that results in minimal CFTR function. In Kaftrio’s triple combination, texacaftor and elexacaftor are designed to increase the amount of properly folded CFTR at the cell surface, while ivacaftor enhances the function of the protein once at the surface.

Kaftrio will also be available to patients with two F508del mutations, giving physicians a new treatment option.

In November 2020, the Transparency Commission (TC) of the French National Authority for Health granted Kaftrio an ASMR 2 rating, which indicated that the therapy provided “a significant improvement in medical service rendered.” Only two medicines have received such a rating out of the 250 issued for first-time treatments in 2019 by the TC, Vertex noted in a press release.

Symkevi (tezacaftor/ivacaftor), marketed as Symdeko in the U.S., will be reimbursed for people with CF 12 years of age and older with one F508del mutation and one mutation resulting in residual CFTR activity, and for those who carry two F508del mutations.

Once the reimbursement agreement has been published in the French Official Journal, both therapies will be available for eligible patients.

“Today’s announcement represents a major milestone for CF patients in France,” said Ludovic Fenaux, senior vice president at Vertex International. “For those living with CF, we are delighted to have reached this agreement so quickly and that the French Health Authorities have recognized the value of both medicines.”

Kaftrio, in combination with ivacaftor, will also be broadly available in Italy to CF patients, ages 12 and older, with one CFTR F508del mutation and one minimal function mutation, or two F508del mutations. Additionally, eligible patients ages 2 and older with two F508del mutations will have access to Orkambi (lumacaftor/ivacaftor), reimbursed for those 12 and older in Italy since 2017. This medicine’s active ingredient, lumacaftor, is designed to target CFTR processing defects.

Broad access to Symkevi plus ivacaftor will also open to eligible patients 12 and older who either carry two copies of the F508del mutation or one F508del mutation plus another residual function mutation. Under the agreement, Kalydeco will be offered to CF patients ages 1 year and older, extending its 2015 addition to Italy’s public health program for those 12 and older.

The Italian agreement also covers approved extensions in use (like changes in eligible age groups or mutations) for Vertex’s CF therapies that are submitted and approved for reimbursement during the contract’s term.

Reimbursement agreements in France and Italy set treatment prices that allow their inclusion in national health programs, through which patients can access prescribed medicines at low, lower, or no cost. Pricing details, or these agreements’ length, were not disclosed.

“This agreement is an important milestone for cystic fibrosis patients in Italy,” Fenaux said in a separate press release. “Our medicines have fundamentally changed the way CF is treated, and we are delighted with this broad portfolio agreement which includes access for younger patients to ORKAMBI and KALYDECO and access to SYMKEVI and KAFTRIO for patients 12 years and older.

“I would like to thank AIFA [the Italian Medicines Agency] and all parties involved for their collaboration, commitment, and engagement in quickly reaching this agreement,” he added.

Vertex noted that its CF medicines are now under reimbursement agreements in more than 25 countries, including the U.S., the U.K., France, Germany, Italy, the Netherlands, Spain, Sweden, the Republic of Ireland, and Australia.