French Authorities Approve Reimbursement of Orkambi for CF Patients 2 Years and Older

The Comité Économique des Produits de Santé (CEPS) in France has approved national reimbursement of Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) for patients with cystic fibrosis (CF) who are 2 years of age or older, with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (the gene defective in CF).

Once the agreement has been published in the French Official Journal, Orkambi will be made available for all eligible CF patients in the country.

In December 2015, Orkambi was made available to CF patients 12 years and older in France through a Temporary Use Authorization program (Autorisations Temporaires d’Utilisation, or ATU), and a post-ATU interim access program.

Temporary Use Authorization is a program that was implemented in France to improve early access to therapies that are under development or already authorized in other countries.

All patients who are currently prescribed Orkambi through the ATU will now be able to receive it through this new agreement.

Importantly, in addition to providing reimbursement for patients 12 years and older, the new agreement will also allow patients 2 to 11 years old in France to access Orkambi for the first time.

“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,” Ludovic Fenaux, senior vice president of Vertex International, said in a press release.

“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.

In related news, Vertex recently set new reimbursement agreements for Orkambi and other CF therapies in England, Wales and Northern Ireland, and other European countries.

Last month, the U.S. the Food and Drug Administration approved a fourth Vertex therapy called Trikafta (elexacaftor, tezacaftor, and ivacaftor) as a next-generation, triple combination treatment for CF. Patients with at least one F508del mutation in the CFTR gene — the most common CF-causing mutation in an estimated 90% of all CF patients — are eligible to receive this treatment.

Vertex has submitted a marketing authorization application to the European Medicines Agency for Trikafta. If approved, further negotiations with national health service agencies will take place.