TRIKAFTA

May 4, 2026: Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) reported Q1 2026 results and highlighted multiple regulatory and clinical catalysts. The company reiterated FY2026 total revenue guidance of $12.95–$13.1B (including ≥$0.5B non-CF product revenue).

Povetacicept (IgA nephropathy; pMN; gMG): After positive Week 36 interim Phase 3 RAINIER data meeting the primary and all secondary endpoints in adults with IgA nephropathy, Vertex completed a rolling BLA submission to the FDA in March for potential U.S. accelerated approval. Vertex expects an expedited 6-month review from BLA acceptance using a Priority Review Voucher. In primary membranous nephropathy, it completed Phase 2 enrollment in the Phase 2/3 OLYMPUS study and initiated Phase 3; FDA Fast Track and Orphan Drug designations and EMA PRIME were noted. A placebo-controlled Phase 2 proof-of-concept study in generalized myasthenia gravis was initiated.

CASGEVY (SCD/TDT): Completed U.S. submission seeking approval in children ages 5 to <12; FDA granted a Commissioner’s National Priority Voucher for accelerated review once accepted.

CF franchise (ALYFTREK/TRIKAFTA): FDA approved label extensions expanding eligible U.S. CF variants; ALYFTREK pediatric (ages 2–5) global submissions targeted in 1H 2026; TRIKAFTA submissions begun for ages 1 to <2.

Pipeline timing updates: VX-828 CF proof-of-concept dosing completion 1H 2026 with results 2H 2026; suzetrigine DPN Phase 3 enrollment completion targeted end-2026; inaxaplin AMKD interim analysis data expected early 2027; VX-670 DM1 results targeted 2H 2026. VX-522 Phase 1/2 ended due to persistent tolerability issues.