UPDATE: Vertex beats own record in cystic fibrosis as next-gen treatment goes up against Trikafta
As a biotechnology industry analyst focusing on cystic fibrosis (CF), I'll provide a factual opinion on the competitive landscape between Vertex's Trikafta and the company's next-gen treatment, vanza (a once-daily triple CFTR modulator consisting of vanzacaftor, tezacaftor, and deutivacaftor).
Trikafta, existing on the market since 2019, is a transformational triple combination therapy approved by the U.S. Food and Drug Administration (FDA) for CF patients aged 2+ who have at least one F508del mutation or specified mutations responsive to the drug (elexacaftor/tezacaftor/ivacaftor). It has shown significant benefits, including a 14% average increase in lung function compared to untreated baselines. Trikafta is now approved for eligible children aged 2 to 5 years.
Recently, Vertex announced Phase 3 trial data for vanza, a new once-daily triple CFTR modulator regimen. The results indicate that vanza has non-inferiority against Trikafta in improving lung function and superiority in reducing sweat chloride levels, a key diagnostic marker for CF, in patients aged 6+ with mutations including F508del. This performance surpassed Trikafta's previous record, with safety profiles showing 95% of patients experiencing treatment-emergent adverse events, primarily infective pulmonary exacerbation of CF.
Vertex intends to file for approval of vanza in the U.S. and Europe for individuals aged 6 and older by mid-2024. Vanza's potential market entry offers an opportunity for patients currently taking Trikafta to achieve even greater CFTR function, complementing Vertex's ongoing CF therapy portfolio evolution.
In summary, the new data suggest that vanza, upon regulatory approval, would offer improved efficacy over Trikafta, with expanded safety profiles that are similar to Trikafta. The development of vanza amplifies Vertex's strength in the CF therapeutics space, providing more personalized treatment options and potentially improving prognoses for CF patients across diverse mutations.