The first triple drug combination therapy - elexacaftor/ivacaftor/tezacaftor (Trikafta) – has been approved by the US FDA, for the treatment of patients 12 years and older with cystic fibrosis, who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which comprises 90% of all patients who have cystic fiborsis.

Mechanism of action: The three drugs in the combination drug target the defective CFTR protein and enable the protein made by the CFTR gene mutation function more effectively.

Adverse effects: Headaches, upper respiratory tract infections, abdominal pains, diarrhea, rashes, increased liver enzymes, nasal congestion, increased creatine phosphokinase, rhinorrhea, rhinitis, influenza, sinusitis and increased blood bilirubin.

Warnings

• Not to be used in patients younger than 12 years
• Raised liver function tests (transaminases and bilirubin)
• Exercise caution when coadministering with drugs that are inducers or inhibitors of cytochrome P450 3A4
• Risk of cataracts

Patients with cystic fibrosis should speak with a health care professional and have tests performed to understand which gene mutations they have. The presence of at least one F508del mutation should be confirmed using an FDA-cleared genotyping assay prior to treatment.

(Source: US FDA)

 Dr KK Aggarwal

Padma Shri Awardee

President Confederation of Medical Associations in Asia and Oceania (CMAAO)

Group Editor-in-Chief IJCP Publications

President Heart Care Foundation of India

Past National President IMA