Although people with these mutations already are approved for Trikafta® in the United States, the study results provide further evidence of the efficacy of the triple-combination therapy for this group and could help inform people's decisions whether to switch modulators. Of course, any decision regarding modulator usage should be discussed with a person's physician.
The study showed a 3.7 percentage point improvement in FEV1 in participants who took Trikafta® compared to their FEV1 after four weeks on ivacaftor (Kalydeco®) for people with an F508del and gating mutation or tezacaftor/ivacaftor (Symdeko®) for people with an F50d8el and residual function mutation. Participants who took Trikafta® also saw a decline in sweat chloride. The treatment was generally well-tolerated and safety data were consistent with those observed in previous Phase 3 studies with Trikafta® with the majority of the adverse events being mild or moderate.
For more information about today's announcement, please see the Vertex press release.
Despite extraordinary progress in helping people with CF live longer and healthier lives, there is still critical work to be done to help all people living with this disease, including those who won't benefit from modulators such as Trikafta®. Through its Path to a Cure, the CF Foundation has dedicated $500 million to develop new treatments for the underlying cause of the disease and, one day, a cure. This initiative centers around three core strategies to address the underlying cause of CF: repairing broken CFTR protein, restoring CFTR protein when none exists, and fixing or replacing the underlying genetic mutation to address the root cause of CF.