Published on
Updated
Reading 2 mins.
Since June 1, the combination of film-coated tablets Kaftrio and Kalydeco has been authorized for new patients with cystic fibrosis, who are not covered by the marketing authorizations (AMM) for these drugs. A victory for the association Vaincre la mucoviscidose.
People affected by cystic fibrosis know it: Kaftrio® is a therapeutic revolution that significantly improves the state of health of eligible patients. If the drug cannot cure the disease, its effectiveness is significant on the respiratory function and therefore the quality of life. However, not all patients can claim it: in France, the Marketing Authorization (MA) for Kaftrio® only concerns patients aged 6 and over, with at least one F508del mutation of the CFTR gene. It is estimated that approximately 3,500 patients are currently treated with Kaftrio®. But an announcement from the National Medicines Agency has just widened access to this treatment.
300 new patients can be treated
Thus, this June 1, 2023, after having “taken into account the evolution of scientific knowledge”, the ANSM has announced a compassionate prescription framework (CPC) combining the drugs Kaftrio and Kalydeco for patients with cystic fibrosis not carrying an F508del mutation, from the age of 6 years and regardless of the degree of severity of the disease.
The decision directly affects 600 patients who, to date, “are not covered by the marketing authorizations (AMM) of these drugs and for which there is an urgent therapeutic need” refers to the ANSM press release. The treatment, on the other hand, would only be effective on half, or 300 patients.
What is the Compassionate Prescribing Framework (CPC)?
The ANSM decides here on a compassionate prescription framework, or CPC, which can frame prescriptions not covered by the marketing authorization (AMM), subject to a therapeutic need and a presumption of benefit report. / favorable risk, in particular based on published scientific data on efficacy and tolerance. The decision, requested by health research institutes or patient associations, is taken for a period of 3 years, renewable.
A world first for Defeat Cystic Fibrosis
For the Vaincre la Mucoviscidose association, research efforts in cystic fibrosis must continue to help all patients. Nevertheless, the ANSM’s announcement is a great step forward and excellent news in this fight.
“The decision taken by the ANSM is a world first since it will allow a large number of patients who do not carry an F508del mutation, regardless of the severity of the disease, to access triple therapy. The joint action of Vaincre la Mucoviscidose and the CRMR Mucoviscidose makes it possible to push Kaftrio® beyond the current Marketing Authorizations and constitutes a tremendous hope for 300 patients!”, underlines David Fiant, president of Vaincre la Mucoviscidose in a press release.