Based on recent studies performed by the CHMP which included clinical trials, lab tests, real world evidence studies & bibliographical data. The European Medicines Agency has recommended to further expand the use of Kaftrio & Kalydeco in combination for treatment of cystic fibrosis in children age starting from two and older who have at least one non-class I mutation in the CFTR gene.
Cystic fibrosis is a serious genetically inherited disease that has a severe effect on lungs, digestive system & other organs. And is known for its high rates of premature mortality. It is caused by alterations in the genes encoding for cystic fibrosis transmembrane conductance regulator (CFTR) protein, which plays a key role in regulating mucus & digestive fluid consistency. Certain mutations impair the function of the CFTR protein, leading to abnormal thick mucus & secretions, which can cause inflammation, blockage, recurrent lung infections, & impaired digestion.
Kaftrio and Kalydeco work by improving the function of faulty CFTR proteins, helping to reduce the thickness of mucus and digestive fluids. These medications, known as CFTR modulators, have already improved outcomes for many patients but weren’t available for all types of mutations until now.
Other mutations like class I mutation doesn’t produce any CFTR proteins hence patients with such mutations don’t respond to the combination therapy using Kaftrio & Kalydeco.
The safety of the Kaftrio and Kalydeco combination remains similar to previous findings, with common side effects like headache, diarrhea, mild infections, and rare cases of serious rash.
The CHMP's positive opinion about this combination is a key step toward wider patient access. It now goes to the European Commission for final approval. After that, each EU country will decide on pricing and reimbursement based on their healthcare system.
