Kaftrio: what is this treatment for cystic fibrosis?

Good news for children with cystic fibrosis! “Kaftrio treatment will be generalized for children with cystic fibrosis” announced François Braun, Minister of Health, to the Journal du Dimanche (JDD) on December 17, 2022.”The decree is ready and should appear in the next few days. The results [du Kaftrio] are extraordinary, they allow an almost ordinary life. It is an immense hope for the 5,500 sick children. It will be available in pharmacies very quickly, on hospital prescription.” added the Minister of Health. Kaftrio® had officially obtained on January 11, 2022 the extension of the European Marketing Authorization for patients aged 6-11 years carrying at least one F508del mutation. “Its marketing should be effective very quickly, after the publication in the Official Journal (JO) of the decree recording its reimbursement. Patients aged 6 years and older who have at least one F508del mutation, regardless of the second mutation, may be prescribed this new disease-modifying treatment by their doctor and order it from a town pharmacy” rejoiced the Association Vaincre la Mucoviscidose in a communicated published on December 17.

What is Kaftrio?

The Kaftrio® is a drug marketed by the Vertex Pharmaceuticals laboratory. It is used for treat patients aged 6 years and over with cystic fibrosis, an inherited disease that has serious effects on the lungs, digestive system and other organs. Kaftrio® received a Marketing authorization valid throughout the European Union on August 21, 2020.

What is the composition of Kaftrio?

Each film-coated tablet contains 75 mg of ivacaftor, 50 mg of tezacaftor and 100 mg of elexacaftor.

What are the indications for Kaftrio?

Kaftrio® is indicated in the treatment of cystic fibrosis in patients aged 6 years and older with at least one F508del mutation in the CFTR gene. The National Agency for the Safety of Medicines and Health Products (ANSM) has established a compassionate prescribing framework (CPC) that allows the drugs Kaftrio® and Kalydeco to be used in combination to treat certain patients with cystic fibrosis who, to date, are not covered by marketing authorizations ( MA) of these drugs and for which there is an urgent therapeutic need. The patients concerned are aged 12 years and over, are not carriers of the F508del mutation and have severe respiratory impairment or a risk of progression to a lung transplant or a life-threatening condition in the more or less short term. Issued in the form of tablets for lifethis triple therapy clearly reduces the effects of the disease, in particular particularly debilitating lung conditions.

How does Kaftrio work?

Cystic fibrosis is caused by mutations in the CFTR gene. This gene leads to the production of the protein CFTR, which acts on the cell surface to regulate the production of mucus in the lungs and digestive juices in the intestine. Mutations reduce the number of CFTR proteins on the cell surface or affect how the protein works. This causes mucus and digestive fluids to become too thick, causing blockages, inflammation, increased risk of lung infections, and poor digestion and growth. Two of the active substances in Kaftrio®elexacaftor and tezacaftor, increase the number of CFTR proteins on the cell surfacewhile the other, ivacaftor, improves the activity of the defective CFTR protein. These actions combine to make lung mucus and digestive juices less thickthus helping to relieve the symptoms of the disease.

What is the dosage of Kaftrio?

Kaftrio® must be taken with another medicine containing ivacaftor alone. The recommended daily dose is two tablets of Kaftrio® at the appropriate dosage in the morning with foods containing fat and one tablet of ivacaftor (150 mg for people receiving the highest dose of Kaftrio®, 75 mg in those receiving the lowest dose) in the evening, approximately 12 hours later. Kaftrio® tablets should be taken with a high fat meal or snack. Meals or snacks high in fat are for example those containing butter or oil or eggs, cheese, nuts, whole milk or meat.

What are the side effects of Kaftrio?

Adverse effects The most common presented by patients are:

• headaches
• diarrhea
• upper respiratory tract infection
• nasopharyngitis
• dizziness
• abdominal pain
• increases in transaminases
• a rash

Common side effects include rhinitis, influenza, hypoglycaemia, earache, abnormal sensation in the ear, tinnitus and hyperaemia.

What are the contraindications and precautions for use of Kaftrio?

Hypersensitivity to the active substances or to any of the excipients is one of the contraindications to taking Kaftrio®. He must also be used with caution in patients with pre-existing advanced liver disease (e.g. cirrhosis, portal hypertension), and only if the expected benefits outweigh the risks. If used, these patients should be closely monitored after initiation of treatment. Treatment is not recommended in patients with moderate hepatic impairment. As a precautionary measure, it is preferable avoid use during pregnancy. If breastfeeding, a decision must be made either to discontinue breast-feeding or to discontinue/abstain from treatment taking into account the benefit of breast-feeding for the child with regard to the benefit of the treatment for the woman. Consumption of food or drink containing grapefruit should be avoided during treatment with Kaftrio®.

Do you need a prescription to get Kaftrio?

The drug is part of the list I of substances that is to say, he is subject to a medical prescription and even a initial hospital prescription.

What is the price of Kaftrio and what reimbursement?

The Kaftrio® costs €10,158.30 and he is reimbursed up to 65%.

Sources:

– Kaftrio: Santa Claus does exist for children, Vaincre la Mucoviscidose, December 17, 2022

– Cystic fibrosis: new patients will be able to benefit from the combination of Kaftrio 75 mg/50 mg/100 mg and Kalydeco 150 mg, ANSM, 05/19/2022

– Kaftrio, European Medicines Agency

– Kafrio, Summary of Product Characteristics, European Commission

– Kaftrio (elexacaftor/ ivacaftor/ tezacaftor) – Cystic fibrosis, HAS, June 8, 2022