Cystic fibrosis (CF) affects more than 30,000 patients in the US. Recent treatments for CF focus on modulating the CFTR gene, with Vertex Pharmaceuticals’ Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor) as the only approved therapies that provide more than symptomatic relief. Those agents, however, either are indicated for small segments of the CF population or offer modest clinical improvements as measured in Equinox Group’s model.
Enter Vertex’s newest treatment, Trikafta (elexacaftor/tezacaftor/ivacaftor), approved in October 2019, which expands the number of CF patients who can benefit from CFTR modulators and offers significant improvement in clinical outcomes. Trikafta has a broad label for all CF patients aged 12 years and older with at least one F508del mutation (approximately 85% of CF patients in the US carry a copy of the F508del mutation). Trikafta’s approval allows Vertex to treat both the underserved heterozygous F508del population and the homozygous F508del population, where both Orkambi and Symdeko are options.
Trikafta offers very high clinical innovation in patients with the heterozygous F508del mutation, delivering a 15% improvement over Pulmozyme (dornase alfa, Genentech) — with clear gains in efficacy, mortality, and morbidity:
For these heterozygous patients, Equinox’s Rare Disease Normative Price Calculator finds Trikafta to be reasonably priced at annual US WAC of $311,500, given its level of clinical benefit.
Equinox Group’s research and predictive model for pricing agents targeted to rare diseases has found that three factors drive pricing potential:
Size of the patient population
Level of disease seriousness (mortality and morbidity), and
Clinical improvement as measured in the Equinox unmet need model
Trikafta’s published data in the homozygous population is not sufficient to allow Equinox to accurately characterize the clinical benefit for those patients, so we cannot comment on the appropriateness of its price in that population.
In Q1 of 2020, Trikafta achieved $900 million in sales. That substantial and rapid commercial success is attributable both to a much larger target population than Vertex’s older CF treatments and to its very high clinical innovation in the heterozygous F508del group.