Vertex has reached an agreement with the French Authorities for a national reimbursement deal of Orkambi, just over a month after securing a similar deal in England following years of access rows.
Orkambi (lumacaftor/ivacaftor) has been approved for national reimbursement in France for people aged two or over with cystic fibrosis, who have two copies of the F508del mutation in the CTFR gene.
After the UK, France has one of the largest CF populations outside the US, with around 2500-3000 patients potentially eligible to benefit from the deal out of the total population.
The treatment has been available in France for eligible patients aged 12 and over since December 2015 through a Temporary Use Authorisation – however, this new deal will see younger patients be able to access the drug for the first time, with all eligible patients fully reimbursed.
“With today’s announcement, eligible patients across France can receive Orkambi through a national reimbursement agreement. Importantly, children with cystic fibrosis as young as two years of age with two copies of the F508del mutation will now benefit from this precision medicine,” said Ludovic Fenaux (pictured below, left), senior vice president, Vertex International.
“We thank the French Authorities for their engagement and commitment that has resulted in an agreement to provide sustainable access to Orkambi for all eligible patients,” he added.
The reimbursement deal with France comes on the heels of another deal, made between Vertex, NHS England and NICE. The American pharma giant reached an agreement with the English regulatory bodies in October, for access to Orkambi and Vertex’s other CF treatments Symkevi (tezacaftor-ivacaftor) and Kalydeco (ivacaftor).
The deal in England was reached after four years of public rows, stalled discussions and frustration for patients, as the three players could not reach an agreement on the pricing of the potentially life-changing therapy.
According to NICE, the negotiation was finalised due to the fact that Vertex finally agreed to the ‘confidential commercial terms’ of the deal – and importantly also agreed to submit its drugs for a full NICE appraisal. The agreement also extends to all future licence extensions of the drugs as well as the current licensed indications.
Vertex is a leader in next-generation CF treatments, with its drugs being the first to address the underlying genetic causes of the disease.
It’s new triple therapy, which is estimated to be effective in around 90% of CF sufferers, has just received approval from the FDA – only three months after it was initially filed with the agency.
Vertex has also submitted the triple therapy to the European Medicines Agency, with a decision expected in 2020.