Breakthrough therapy for cystic fibrosis

Trikafka™ was approved five months earlier than expected and launched the last week of October 2019. Its triple combination therapy (elexacaftor/ivacaftor/tezacaftor) may cover 90% of patients with cystic fibrosis.

Condition overview

About 30,000 people in the U.S. have cystic fibrosis. This is a progressive genetic disease that damages the lungs and digestive system. It’s caused by mutations in a specific gene, the cystic fibrosis transmembrane conductance regulator (CFTR) gene.1 Mutations in that gene cause the buildup of mucus in various organs. In the lungs, that mucus traps germs, causing infections and respiratory distress. In the pancreas, the mucus blocks the absorption of nutrients, causing malnutrition and poor growth. In the liver, the mucus clogs the bile duct, causing liver disease.1

People with CF may have a range of symptoms, including:

• Persistent coughing, at times with phlegm
• Frequent lung infections including pneumonia or bronchitis
• Wheezing or shortness of breath
• Poor growth or weight gain in spite of a good appetite
• Frequent greasy, bulky stools or difficulty with bowel movements
• There are more than 1,700 different mutations associated with cystic fibrosis. Treatments that work for some may mutations may not work for others.

Current treatments

For most people with cystic fibrosis, maintaining lung function and avoiding infections are their highest priorities.3 Patients with cystic fibrosis generally use a combination of the following therapies:

• Airway clearance to help loosen and get rid of the thick mucus that can build up in the lungs (airway clearance techniques, chest physical therapy, coughing and huffing, active cycle of breathing technique, autogenic drainage, positive expiratory pressure, high-frequency chest wall oscillation (the vest).
• Inhaled medicines to open the airways or thin the mucus. These are liquid medicines that are made into a mist or aerosol and then inhaled through a nebulizer. They include antibiotics to fight lung infections and therapies to help keep the airways clear.
• Pancreatic enzyme supplement capsules to improve the absorption of vital nutrients. These supplements are taken with every meal and most snacks. People with CF also usually take multivitamins.
• An individualized fitness plan to help improve energy, lung function, and overall health.

CFTR modulators to target the underlying defect in the CFTR protein. Because different mutations cause different defects in the protein, the medications that have been developed so far are effective only in people with specific mutations.

Overview of Trikafka

Trikafka is indicated for patients over 12 years old with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Trikafka targets the protein made by the CFTR gene mutation and helps it function more effectively.

This indication covers approximately 18,000 CF patients in the United States:

• 6,000 who were not eligible for one of Vertex’s three FDA-approved CF medications [Kalydeco® (ivacaftor), Symdeko® (tezacaftor/ivacaftor and ivacaftor), and Orkambi® (lumacaftor/ivacaftor)]; and
• Approximately 12,000 CF patients who are currently eligible for one of the three Vertex drugs.1

Together, this represents 90% of the cystic fibrosis population.2

Date approved: October 21, 2019
Benefit: Trikafka will be covered under the pharmacy benefit. It is an oral formulation.

Prime monitors the drug pipeline

The drug pipeline is full of new, groundbreaking specialty drugs that may help members feel better and live well. Prime focuses on clinical strategies designed to keep clients ahead of drug trends — because it’s easier to manage change when you see it coming.

References

1. https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/
2. https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis
3. https://www.cff.org/Life-With-CF/Treatments-and-Therapies/Airway-Clearance/