Vertex Pharmaceuticals (VRTX) investor relations material

Strategic priorities and portfolio evolution

• Expanded from a single franchise in cystic fibrosis to three, with launches in pain and sickle cell, and a fourth in renal on the horizon.

• Focus on extending leadership in cystic fibrosis, building blockbuster franchises in hematology (CASGEVY) and pain (JOURNAVX), and establishing a substantial renal vertical.

• Emphasis on tech-enabled workforce and responsible AI integration to support innovation and product pipeline.

• Geographic expansion includes new markets such as the Middle East, with CASGEVY launched in Riyadh in 2023.

• Preparing for future launches in type 1 diabetes with cell-based therapies, requiring significant commercial and manufacturing buildout.

Policy, regulatory, and market environment

• Relatively insulated from recent policy risks like MFN and GloBE due to product mix and rare disease carve-outs.

• Diverse supply chain and significant U.S. manufacturing mitigate tariff impacts, with no expected effect from tariffs in 2026.

• Ongoing FDA interactions remain smooth despite broader agency disruptions; filings and meetings continue within expected timelines.

Cystic fibrosis franchise performance and outlook

• ALYFTREK, the fifth CF medicine, is now the emerging standard of care, covering 95% of eligible patients and offering once-daily dosing.

• Launches have been particularly strong outside the U.S. due to broader mutation coverage and less stringent monitoring requirements.

• Growth drivers include increased patient longevity, expansion to younger age groups, geographic expansion, and higher eligibility rates.

• Franchise durability is supported by strong real-world efficacy, emotional patient attachment, and long-term registry data.

• Focus shifting to surpassing ALYFTREK with next-generation therapies as measurement endpoints reach their limits.