Vertex Pharmaceuticals (VRTX) investor relations material

Commercial performance and product launches

• Acute pain launch achieved over 170 million covered lives in the U.S. within 10 months, with more than 300,000 prescriptions written and strong feedback from physicians and payers.

• Alyftrek generated nearly $500 million in revenue in its first nine months, with rapid uptake among naive and previously discontinued patients, and steady switching from older therapies.

• Alyftrek's European label lacks liver monitoring requirements, leading to faster uptake, especially among patients with rare mutations.

• Casgevy is expected to surpass $100 million in revenue in 2025, with a growing patient funnel and significant ramp anticipated in 2026.

• Access and reimbursement for Casgevy are strong in the U.S., Middle East, and major European markets.

Clinical development and pipeline progress

• Focus on diabetic peripheral neuropathy (DPN) with two Phase 3 studies enrolling, aiming for completion in 2026, addressing a 2 million patient U.S. market.

• Selective sodium channel inhibitors NaV1.8 and NaV1.7 are being advanced, with NaV1.8 already approved and NaV1.7 in preclinical development.

• Early treatment in cystic fibrosis shows promise for long-term benefits, with ongoing studies in very young children and efforts to address rare mutations through mRNA and gene therapy collaborations.

• POVItacicept (POVI) is advancing rapidly in renal and B-cell mediated diseases, with interim analysis data expected soon and rolling submissions underway.

• APOL1-mediated kidney disease program is in Phase 3, with interim data expected in late 2026, targeting a genetically validated population.

Market access, policy, and regulatory environment

• Progress with payers and PBMs continues, with ongoing efforts to expand Medicare access and address policy issues like the No Pain Act.

• New reimbursement discussions for Alyftrek are required in many European countries, potentially affecting launch timelines.

• Regulatory enthusiasm in nephrology is high, with proteinuria recognized as a predictive marker, facilitating accelerated approvals.