FAQs about CFTR modulators

Category: CFTR modulators

CFTR modulators are not considered a cure for cystic fibrosis (CF), though the medications can help to ease CF symptoms and slow disease progression in people with specific disease-causing mutations. While CFTR modulators can increase the functionality of mutated CFTR protein, they don’t completely normalize the protein’s activity, so patients may still experience some symptoms and progression even with modulator treatment. 

Category: CFTR modulators

Cystic fibrosis (CF) is caused by mutations that impair the functionality of the CFTR protein. CFTR modulators are medications that can bind to specific mutated versions of this protein, increasing its function. By boosting the function of the CFTR protein, CFTR modulators can help to ease symptoms of CF and slow disease progression in patients with amenable mutations.

Category: CFTR modulators

All four CFTR modulators now available — Kalydeco, Orkambi, Symdeko, and Trikafta — are taken by mouth, twice daily, with varying dosages depending on the patient’s age and weight. Depending on the cystic fibrosis medication and formulation, these therapies may be available in the form of granules or tablets. Each dose of modulator therapy should be taken along with a fat-containing food (e.g., eggs, meat, nuts, or whole-milk dairy products), and foods containing grapefruit should be avoided.

Category: CFTR modulators

CFTR modulators may cause side effects that include cold-like symptoms (runny nose, congestion, etc.) and gastrointestinal effects — nausea, diarrhea, and abdominal pain — as well as headache, dizziness, rash, infections, cataracts, and liver damage. The specific side effect profile varies depending on the particular cystic fibrosis medication; patients are advised to consult with their healthcare providers about any effects they experience after starting on CFTR modulator therapy.

Category: CFTR modulators

The CFTR protein normally functions like a gate at the surface of a cell, helping to regulate the flow of chloride in and out of the cell. CFTR potentiators are a type of modulator therapy that have the effect of propping open the gate-like protein at the cell surface. CFTR correctors are a distinct type of therapy that helps stabilize the CFTR protein so it can get to the cell surface without being degraded.