Vertex Pharmaceuticals expands pipeline beyond cystic fibrosis

Vertex Pharmaceuticals is working to diversify its revenue stream, which currently relies on cystic fibrosis (CF) therapies for approximately 96% of its sales. While the company has seen significant success with CFTR modulators like Trikafta and Alyftrek, it is now advancing several new programs.

Key developments include the gene-editing therapy Casgevy, developed with CRISPR Therapeutics for sickle cell disease and beta-thalassemia, and the non-opioid pain reliever Journavx, which is expected to generate $500 million or more in sales by 2026.

Additionally, the company reported positive Phase 2b trial data for inaxaplin, an oral therapy for APOL1-mediated kidney disease (AKMD). The trial indicated a reduction in protein leaking in urine without serious side effects. Vertex is also presenting updates on povetacicept for IgA nephropathy (IgAN) at the American Society of Nephrology Kidney Week 2026.