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The ANSM has just authorized access to Kaftrio® to patients with cystic fibrosis who could not benefit from this treatment until now. 600 patients will benefit from this compassionate protocol program, which should improve the health of half of them.

Kaftrio® is a treatment that significantly improves the state of health of patients with cystic fibrosis. If it does not cure the disease, it allows patients to live almost normally, reducing its respiratory effects. In France, the Marketing Authorization (AMM) for this drug now concerns patients 6 years of age and older with at least one F508del mutation in the CFTR gene. It is estimated that around 3,500 patients can benefit from this treatment in France.

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On the other hand, until now, patients who did not carry the gene mutation were excluded from this Marketing Authorisation. Only a few patients with “severe respiratory impairment or a risk of progression to a lung transplant or a vital prognosis committed in the more or less short term” have been able to benefit from this treatment since May 2022, as part of a compassionate prescription ( CPC).

Immediate severity criteria are removed

“About 120 patients have benefited from this CBD program with proven efficacy in about 60 patients” underlines the Vaincre la Mucoviscidose association, which funded this study conducted by the teams of Pr Pierre-Régis Burgel (Cochin Hospital – Port Royal AP -HP) and Isabelle Sermet-Gaudelus (Necker Hospital – AP-HP sick children). “Given these very encouraging results, Vaincre la Mucoviscidose and the Rare Disease Reference Center coordinator Mucoviscidose (CRMR) have requested an extension of the compassionate prescription framework” adds the association.

This request has been heard and the ANSM has just responded favorably by eliminating the need to have immediate severity criteria to benefit from Kaftrio®. This decision should make it possible in the coming weeks to administer Kaftrio® to nearly 600 patients under a temporary use protocol for a period of 2 months.

600 new patients will be able to test the treatment

“After an initial period of treatment with Kaftrio® of 4 to 6 weeks, the efficacy of the treatment being rapid, it may be decided to continue the treatment if it is deemed effective or to discontinue it if the beneficial effects are deemed In view of the studies previously carried out, the treatment should prove effective for approximately 300 patients” specifies Professor Pierre-Régis Burgel.

CPC Kaftrio® now concerns:

  • patients with cystic fibrosis aged 6 years and older
  • not carrying an F508del mutation excluding mutations not producing CFTR protein
  • regardless of the severity of the disease.

“The result is up to our expectations, but we are looking forward to access for patients aged 2 to 5 yearsfor which an MA is currently being examined by the European Medicines Agency and we hope that the laboratory will initiate an early access request this summer” insists David Fiant, president of Vaincre la Mucoviscidose.

The most common serious genetic disease

Cystic fibrosis is the most common serious genetic disease from childhood: in 2020, 108 children with cystic fibrosis were born in France. This disease is characterized by mutations in the gene encoding the protein CFTR which regulates the transport of chlorine through the membranes of the bodily glandular mucous membranes. If this protein malfunctions, the sweat is abnormally salty and the mucous secretions abnormally viscous, leading in particular to obstructions or secondary infections in the bronchi and pancreas.

Source : Cystic fibrosis: new patients will be able to benefit from the combination of drugs Kaftrio 75 mg/50 mg/100 mg and kalydeco 150 mg, ANSM, June 2023

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