Following the news that Vertex’s Trikafta/Kaftrio has been approved for children ages six to 11 years with cystic fibrosis (CF);

Mandana Emamzadeh, PhD, Healthcare Analyst at GlobalData, a leading data and analytics company, offers her view:

“Trikafta/Kaftrio has greatly expanded the number of CF-related mutations that are eligible for CFTR modulator treatment across age groups. The drug is reported to decrease the use of symptomatic drug classes such as antibiotics, mucolytics, and anti-inflammatory drugs due to overall improvements to lung health in patients. As a result, the overall survival of CF patients is expected to increase remarkably over the next decades.

“Children born today with CF have a more optimistic prognosis compared to children born more than 30 years ago, with the majority of CF patients in the US and UK now expected to live beyond their late 40s. According to the Cystic Fibrosis Foundation (CFF) and the Cystic Fibrosis Trust (CFT), most CF patients reach adulthood with currently available treatments. Further, key opinion leaders interviewed by GlobalData asserted that for patients born in the past decade, the median survival is expected to be more than 50 years of age.

“Trikafta/Kaftrio is currently the world’s first triple-combination therapy for which approximately 90% of the CF population will likely be eligible. Additionally, Vertex Pharmaceuticals is investigating Trikafta/Kaftrio in Phase III for CF patients ages two to five years to expand the label for its game-changer drug in the seven major markets (7MM). If successful, Trikafta/Kaftrio will be available for the majority of CF-related mutations across all age groups. GlobalData estimates that Trikafta’s global peak sales will reach $7.2 billion in 2024, after which sales will decline due to competition from other CFTR modulators.”

*7MM: The US, France, Germany, Italy, Spain, the UK, and Japan